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Product approved to treat patients with hemophilia A and inhibitors
The US Food and Drug Administration (FDA) has approved use of emicizumab-kxwh (Hemlibra®), a bispecific factor IXa- and factor X-directed antibody.
Emicizumab is approved as routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children who have hemophilia A and factor VIII (FVIII) inhibitors.
Emicizumab can be self-administered once-weekly via subcutaneous injection.
The labeling for emicizumab contains a boxed warning noting that patients who received emicizumab in conjunction with activated prothrombin complex concentrate developed thrombotic microangiopathy (TMA) and thromboembolic events (TEs).
Therefore, patients should discontinue prophylactic use of bypassing agents (BPAs) the day before starting prophylaxis with emicizumab.
The FDA granted the approval of emicizumab to Genentech, Inc. The agency granted the application for emicizumab priority review, and the product received breakthrough therapy and orphan drug designations.
Access to emicizumab
According to Genentech, emicizumab will be available shortly.
The company said it will be offering comprehensive services to help minimize barriers to access and reimbursement. Patients can call 866-436-5427 (866-HEMLIBRA) for more information.
For people who qualify, Genentech plans to offer patient assistance programs through Genentech Access Solutions. More information is available at 866-422-2377 (866-4ACCESS) or http://www.Genentech-Access.com.
Emicizumab trials
The biologics license application for emicizumab was supported by results from a pair of phase 3 studies—HAVEN 1 and HAVEN 2.
Results from HAVEN 1 were published in NEJM and presented at the 26th ISTH Congress in July. Interim results from HAVEN 2 were presented at ISTH as well.
HAVEN 1
The study enrolled 109 patients (age 12 and older) with hemophilia A and FVIII inhibitors who were previously treated with BPAs on-demand or as prophylaxis.
The patients were randomized to receive emicizumab prophylaxis or no prophylaxis. On-demand treatment of breakthrough bleeds with BPAs was allowed.
There was a significant reduction in treated bleeds of 87% with emicizumab prophylaxis compared to no prophylaxis (95% CI: 72.3; 94.3, P<0.0001). And there was an 80% reduction in all bleeds with emicizumab (95% CI: 62.5; 89.8, P<0.0001).
Adverse events occurring in at least 5% of patients treated with emicizumab were local injection site reactions, headache, fatigue, upper respiratory tract infection, and arthralgia.
Two patients experienced TEs, and 3 had TMA while receiving emicizumab prophylaxis and more than 100 u/kg/day of activated prothrombin complex concentrate, on average, for 24 hours or more before the event. Two of these patients had also received recombinant factor VIIa.
Neither TE required anticoagulation therapy, and 1 patient restarted emicizumab. The cases of TMA observed were transient, and 1 patient restarted emicizumab.
HAVEN 2
In this single-arm trial, researchers evaluated emicizumab prophylaxis in children younger than 12 years of age who had hemophilia A with FVIII inhibitors.
The interim efficacy analysis, after at least 12 weeks of treatment, included 23 children.
After a median observation time of 38.1 weeks, 87% (95% CI: 66.4; 97.2) of children who received emicizumab experienced 0 treated bleeds. The percentage with 0 treated or non-treated bleeds was lower, at 34.8% (95% CI: 16.4; 57.3).
The most common adverse events (observed in at least 10% of patients) were mild injection site reactions and nasopharyngitis. No TEs or TMAs were observed.
HAVEN 3 and 4
Emicizumab is now being studied in 2 additional phase 3 trials.
In HAVEN 3, researchers are evaluating emicizumab prophylaxis dosed once weekly or once every other week in patients age 12 and older with hemophilia A without FVIII inhibitors.
In HAVEN 4, researchers are evaluating emicizumab prophylaxis dosed every 4 weeks in patients age 12 and older with hemophilia A, with or without inhibitors. 
The US Food and Drug Administration (FDA) has approved use of emicizumab-kxwh (Hemlibra®), a bispecific factor IXa- and factor X-directed antibody.
Emicizumab is approved as routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children who have hemophilia A and factor VIII (FVIII) inhibitors.
Emicizumab can be self-administered once-weekly via subcutaneous injection.
The labeling for emicizumab contains a boxed warning noting that patients who received emicizumab in conjunction with activated prothrombin complex concentrate developed thrombotic microangiopathy (TMA) and thromboembolic events (TEs).
Therefore, patients should discontinue prophylactic use of bypassing agents (BPAs) the day before starting prophylaxis with emicizumab.
The FDA granted the approval of emicizumab to Genentech, Inc. The agency granted the application for emicizumab priority review, and the product received breakthrough therapy and orphan drug designations.
Access to emicizumab
According to Genentech, emicizumab will be available shortly.
The company said it will be offering comprehensive services to help minimize barriers to access and reimbursement. Patients can call 866-436-5427 (866-HEMLIBRA) for more information.
For people who qualify, Genentech plans to offer patient assistance programs through Genentech Access Solutions. More information is available at 866-422-2377 (866-4ACCESS) or http://www.Genentech-Access.com.
Emicizumab trials
The biologics license application for emicizumab was supported by results from a pair of phase 3 studies—HAVEN 1 and HAVEN 2.
Results from HAVEN 1 were published in NEJM and presented at the 26th ISTH Congress in July. Interim results from HAVEN 2 were presented at ISTH as well.
HAVEN 1
The study enrolled 109 patients (age 12 and older) with hemophilia A and FVIII inhibitors who were previously treated with BPAs on-demand or as prophylaxis.
The patients were randomized to receive emicizumab prophylaxis or no prophylaxis. On-demand treatment of breakthrough bleeds with BPAs was allowed.
There was a significant reduction in treated bleeds of 87% with emicizumab prophylaxis compared to no prophylaxis (95% CI: 72.3; 94.3, P<0.0001). And there was an 80% reduction in all bleeds with emicizumab (95% CI: 62.5; 89.8, P<0.0001).
Adverse events occurring in at least 5% of patients treated with emicizumab were local injection site reactions, headache, fatigue, upper respiratory tract infection, and arthralgia.
Two patients experienced TEs, and 3 had TMA while receiving emicizumab prophylaxis and more than 100 u/kg/day of activated prothrombin complex concentrate, on average, for 24 hours or more before the event. Two of these patients had also received recombinant factor VIIa.
Neither TE required anticoagulation therapy, and 1 patient restarted emicizumab. The cases of TMA observed were transient, and 1 patient restarted emicizumab.
HAVEN 2
In this single-arm trial, researchers evaluated emicizumab prophylaxis in children younger than 12 years of age who had hemophilia A with FVIII inhibitors.
The interim efficacy analysis, after at least 12 weeks of treatment, included 23 children.
After a median observation time of 38.1 weeks, 87% (95% CI: 66.4; 97.2) of children who received emicizumab experienced 0 treated bleeds. The percentage with 0 treated or non-treated bleeds was lower, at 34.8% (95% CI: 16.4; 57.3).
The most common adverse events (observed in at least 10% of patients) were mild injection site reactions and nasopharyngitis. No TEs or TMAs were observed.
HAVEN 3 and 4
Emicizumab is now being studied in 2 additional phase 3 trials.
In HAVEN 3, researchers are evaluating emicizumab prophylaxis dosed once weekly or once every other week in patients age 12 and older with hemophilia A without FVIII inhibitors.
In HAVEN 4, researchers are evaluating emicizumab prophylaxis dosed every 4 weeks in patients age 12 and older with hemophilia A, with or without inhibitors.
The US Food and Drug Administration (FDA) has approved use of emicizumab-kxwh (Hemlibra®), a bispecific factor IXa- and factor X-directed antibody.
Emicizumab is approved as routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children who have hemophilia A and factor VIII (FVIII) inhibitors.
Emicizumab can be self-administered once-weekly via subcutaneous injection.
The labeling for emicizumab contains a boxed warning noting that patients who received emicizumab in conjunction with activated prothrombin complex concentrate developed thrombotic microangiopathy (TMA) and thromboembolic events (TEs).
Therefore, patients should discontinue prophylactic use of bypassing agents (BPAs) the day before starting prophylaxis with emicizumab.
The FDA granted the approval of emicizumab to Genentech, Inc. The agency granted the application for emicizumab priority review, and the product received breakthrough therapy and orphan drug designations.
Access to emicizumab
According to Genentech, emicizumab will be available shortly.
The company said it will be offering comprehensive services to help minimize barriers to access and reimbursement. Patients can call 866-436-5427 (866-HEMLIBRA) for more information.
For people who qualify, Genentech plans to offer patient assistance programs through Genentech Access Solutions. More information is available at 866-422-2377 (866-4ACCESS) or http://www.Genentech-Access.com.
Emicizumab trials
The biologics license application for emicizumab was supported by results from a pair of phase 3 studies—HAVEN 1 and HAVEN 2.
Results from HAVEN 1 were published in NEJM and presented at the 26th ISTH Congress in July. Interim results from HAVEN 2 were presented at ISTH as well.
HAVEN 1
The study enrolled 109 patients (age 12 and older) with hemophilia A and FVIII inhibitors who were previously treated with BPAs on-demand or as prophylaxis.
The patients were randomized to receive emicizumab prophylaxis or no prophylaxis. On-demand treatment of breakthrough bleeds with BPAs was allowed.
There was a significant reduction in treated bleeds of 87% with emicizumab prophylaxis compared to no prophylaxis (95% CI: 72.3; 94.3, P<0.0001). And there was an 80% reduction in all bleeds with emicizumab (95% CI: 62.5; 89.8, P<0.0001).
Adverse events occurring in at least 5% of patients treated with emicizumab were local injection site reactions, headache, fatigue, upper respiratory tract infection, and arthralgia.
Two patients experienced TEs, and 3 had TMA while receiving emicizumab prophylaxis and more than 100 u/kg/day of activated prothrombin complex concentrate, on average, for 24 hours or more before the event. Two of these patients had also received recombinant factor VIIa.
Neither TE required anticoagulation therapy, and 1 patient restarted emicizumab. The cases of TMA observed were transient, and 1 patient restarted emicizumab.
HAVEN 2
In this single-arm trial, researchers evaluated emicizumab prophylaxis in children younger than 12 years of age who had hemophilia A with FVIII inhibitors.
The interim efficacy analysis, after at least 12 weeks of treatment, included 23 children.
After a median observation time of 38.1 weeks, 87% (95% CI: 66.4; 97.2) of children who received emicizumab experienced 0 treated bleeds. The percentage with 0 treated or non-treated bleeds was lower, at 34.8% (95% CI: 16.4; 57.3).
The most common adverse events (observed in at least 10% of patients) were mild injection site reactions and nasopharyngitis. No TEs or TMAs were observed.
HAVEN 3 and 4
Emicizumab is now being studied in 2 additional phase 3 trials.
In HAVEN 3, researchers are evaluating emicizumab prophylaxis dosed once weekly or once every other week in patients age 12 and older with hemophilia A without FVIII inhibitors.
In HAVEN 4, researchers are evaluating emicizumab prophylaxis dosed every 4 weeks in patients age 12 and older with hemophilia A, with or without inhibitors.
FDA expands approval of obinutuzumab
The US Food and Drug Administration (FDA) has expanded the approved use of obinutuzumab (Gazyva®).
The drug is now approved for use in combination with chemotherapy to treat patients with previously untreated follicular lymphoma (FL) that is advanced (stage II bulky, stage III, or stage IV) .
In patients who respond to this treatment, obinutuzumab monotherapy can be given as maintenance.
The FDA granted this new approval of obinutuzumab to Genentech, Inc. The application for obinutuzumab in this indication received priority review.
The latest FDA approval means obinutuzumab is available in the US for the following indications:
- In combination with chlorambucil to treat chronic lymphocytic leukemia (CLL) in adults who have not had previous CLL treatment
- In combination with bendamustine, followed by obinutuzumab alone, to treat FL in adults who did not respond to a rituximab-containing regimen or whose FL returned after such treatment
- In combination with chemotherapy, followed by obinutuzumab alone in responders, to treat stage II bulky, stage III, or stage IV FL in adults who have not had previous FL treatment.
Phase 3 results
The latest approval of obinutuzumab is based on results from the phase 3 GALLIUM study, which were presented at the 2016 ASH Annual Meeting and published in NEJM in October.
The following are updated data from the obinutuzumab prescribing information.
GALLIUM included 1385 patients with previously untreated non-Hodgkin lymphoma, and 1202 of these patients had advanced FL.
Half of the FL patients (n=601) were randomized to receive obinutuzumab plus chemotherapy (followed by obinutuzumab maintenance for up to 2 years), and half were randomized to rituximab plus chemotherapy (followed by rituximab maintenance for up to 2 years).
The different chemotherapies used were CHOP (cyclophosphamide, doxorubicin, vincristine, and prednisolone), CVP (cyclophosphamide, vincristine, and prednisolone), and bendamustine.
At a median observation time of 38 months, the overall response rate was 91% in the obinutuzumab arm and 88% in the rituximab arm. The complete response rates were 28% and 27%, respectively.
The median progression-free survival was not reached in either arm. The hazard ratio, for obinutuzumab compared to rituximab, was 0.72 (95% CI, 0.56-0.93, P=0.0118).
Safety was evaluated based on all 1385 patients in the study, 86% of whom had previously untreated FL and 14% of whom had marginal zone lymphoma.
Serious adverse events (AEs) occurred in 50% of patients in the obinutuzumab arm and 43% in the rituximab arm. Fatal AEs occurred in 5% and 4%, respectively. Infections and second malignancies were the leading causes of these deaths.
The most common AEs (incidence ≥ 20%) observed at least 2% more patients in the obinutuzumab arm were infusion-related reactions, neutropenia, upper respiratory tract infection, cough, constipation, and diarrhea.
The most common grade 3 to 5 AEs (incidence ≥ 5%) observed more frequently in the obinutuzumab arm were neutropenia, infusion-related reactions, febrile neutropenia, and thrombocytopenia.
The US Food and Drug Administration (FDA) has expanded the approved use of obinutuzumab (Gazyva®).
The drug is now approved for use in combination with chemotherapy to treat patients with previously untreated follicular lymphoma (FL) that is advanced (stage II bulky, stage III, or stage IV) .
In patients who respond to this treatment, obinutuzumab monotherapy can be given as maintenance.
The FDA granted this new approval of obinutuzumab to Genentech, Inc. The application for obinutuzumab in this indication received priority review.
The latest FDA approval means obinutuzumab is available in the US for the following indications:
- In combination with chlorambucil to treat chronic lymphocytic leukemia (CLL) in adults who have not had previous CLL treatment
- In combination with bendamustine, followed by obinutuzumab alone, to treat FL in adults who did not respond to a rituximab-containing regimen or whose FL returned after such treatment
- In combination with chemotherapy, followed by obinutuzumab alone in responders, to treat stage II bulky, stage III, or stage IV FL in adults who have not had previous FL treatment.
Phase 3 results
The latest approval of obinutuzumab is based on results from the phase 3 GALLIUM study, which were presented at the 2016 ASH Annual Meeting and published in NEJM in October.
The following are updated data from the obinutuzumab prescribing information.
GALLIUM included 1385 patients with previously untreated non-Hodgkin lymphoma, and 1202 of these patients had advanced FL.
Half of the FL patients (n=601) were randomized to receive obinutuzumab plus chemotherapy (followed by obinutuzumab maintenance for up to 2 years), and half were randomized to rituximab plus chemotherapy (followed by rituximab maintenance for up to 2 years).
The different chemotherapies used were CHOP (cyclophosphamide, doxorubicin, vincristine, and prednisolone), CVP (cyclophosphamide, vincristine, and prednisolone), and bendamustine.
At a median observation time of 38 months, the overall response rate was 91% in the obinutuzumab arm and 88% in the rituximab arm. The complete response rates were 28% and 27%, respectively.
The median progression-free survival was not reached in either arm. The hazard ratio, for obinutuzumab compared to rituximab, was 0.72 (95% CI, 0.56-0.93, P=0.0118).
Safety was evaluated based on all 1385 patients in the study, 86% of whom had previously untreated FL and 14% of whom had marginal zone lymphoma.
Serious adverse events (AEs) occurred in 50% of patients in the obinutuzumab arm and 43% in the rituximab arm. Fatal AEs occurred in 5% and 4%, respectively. Infections and second malignancies were the leading causes of these deaths.
The most common AEs (incidence ≥ 20%) observed at least 2% more patients in the obinutuzumab arm were infusion-related reactions, neutropenia, upper respiratory tract infection, cough, constipation, and diarrhea.
The most common grade 3 to 5 AEs (incidence ≥ 5%) observed more frequently in the obinutuzumab arm were neutropenia, infusion-related reactions, febrile neutropenia, and thrombocytopenia.
The US Food and Drug Administration (FDA) has expanded the approved use of obinutuzumab (Gazyva®).
The drug is now approved for use in combination with chemotherapy to treat patients with previously untreated follicular lymphoma (FL) that is advanced (stage II bulky, stage III, or stage IV) .
In patients who respond to this treatment, obinutuzumab monotherapy can be given as maintenance.
The FDA granted this new approval of obinutuzumab to Genentech, Inc. The application for obinutuzumab in this indication received priority review.
The latest FDA approval means obinutuzumab is available in the US for the following indications:
- In combination with chlorambucil to treat chronic lymphocytic leukemia (CLL) in adults who have not had previous CLL treatment
- In combination with bendamustine, followed by obinutuzumab alone, to treat FL in adults who did not respond to a rituximab-containing regimen or whose FL returned after such treatment
- In combination with chemotherapy, followed by obinutuzumab alone in responders, to treat stage II bulky, stage III, or stage IV FL in adults who have not had previous FL treatment.
Phase 3 results
The latest approval of obinutuzumab is based on results from the phase 3 GALLIUM study, which were presented at the 2016 ASH Annual Meeting and published in NEJM in October.
The following are updated data from the obinutuzumab prescribing information.
GALLIUM included 1385 patients with previously untreated non-Hodgkin lymphoma, and 1202 of these patients had advanced FL.
Half of the FL patients (n=601) were randomized to receive obinutuzumab plus chemotherapy (followed by obinutuzumab maintenance for up to 2 years), and half were randomized to rituximab plus chemotherapy (followed by rituximab maintenance for up to 2 years).
The different chemotherapies used were CHOP (cyclophosphamide, doxorubicin, vincristine, and prednisolone), CVP (cyclophosphamide, vincristine, and prednisolone), and bendamustine.
At a median observation time of 38 months, the overall response rate was 91% in the obinutuzumab arm and 88% in the rituximab arm. The complete response rates were 28% and 27%, respectively.
The median progression-free survival was not reached in either arm. The hazard ratio, for obinutuzumab compared to rituximab, was 0.72 (95% CI, 0.56-0.93, P=0.0118).
Safety was evaluated based on all 1385 patients in the study, 86% of whom had previously untreated FL and 14% of whom had marginal zone lymphoma.
Serious adverse events (AEs) occurred in 50% of patients in the obinutuzumab arm and 43% in the rituximab arm. Fatal AEs occurred in 5% and 4%, respectively. Infections and second malignancies were the leading causes of these deaths.
The most common AEs (incidence ≥ 20%) observed at least 2% more patients in the obinutuzumab arm were infusion-related reactions, neutropenia, upper respiratory tract infection, cough, constipation, and diarrhea.
The most common grade 3 to 5 AEs (incidence ≥ 5%) observed more frequently in the obinutuzumab arm were neutropenia, infusion-related reactions, febrile neutropenia, and thrombocytopenia.
CAR T-cell therapy on fast track with FDA, EMA
A chimeric antigen receptor (CAR) T-cell therapy, bb2121, has received breakthrough therapy designation from the US Food and Drug Administration (FDA) and was granted access to the European Medicines Agency’s (EMA’s) PRIority MEdicines (PRIME) program.
The CAR T-cell therapy is designed to target B-cell maturation antigen in previously treated patients with multiple myeloma.
bb2121 is being developed by bluebird bio, Inc., and Celgene Corporation.
The EMA’s and FDA’s decisions on bb2121 were based on preliminary data from an ongoing phase 1 study, CRB-401 (NCT02658929).
Results from this study were presented at the 2017 ASCO Annual Meeting (abstract 3010).
The study enrolled patients with relapsed and/or refractory multiple myeloma. As of the May 4, 2017 data cut-off, 21 patients had been enrolled.
Patients had a median of 7 prior lines of therapy (range, 3-14). Their previous treatments included lenalidomide and bortezomib (100%), pomalidomide and carfilzomib (91%), daratumumab (71%), and autologous stem cell transplant (100% at least once).
Twenty-nine percent of patients were refractory to bortezomib, lenalidomide, carfilzomib, pomalidomide, and daratumumab.
Patients received a conditioning regimen of cyclophosphamide and fludarabine, followed by an infusion of bb2121 at 1 of 4 doses: 50 x 106, 150 x 106, 450 x 106 and 800 x 106 CAR+ T cells.
All 21 patients were evaluable for safety.
The most common treatment-emergent grade 3-4 adverse events were cytopenias commonly associated with the lymphodepletion regimen, as well as grade 3 events of hyponatremia (n=4), upper respiratory infection (n=2), syncope (n=2), and cytokine release syndrome (CRS, n=2).
In all, 71% of patients (15/21) had CRS, mostly grade 1 and 2. For the 2 patients with grade 3 CRS, it resolved within 24 hours. To manage CRS, 4 patients received tocilizumab, and 1 (with grade 2) received steroids as well.
Eighteen patients were evaluable for efficacy, and the overall response rate was 89% (16/18).
There were 4 complete responses—2 in the 150 x 106 cohort, 1 in the 450 x 106 cohort, and 1 in the 800 x 106 cohort. The complete responder in the 450 x 106 cohort ultimately died of cardiopulmonary arrest that was deemed unrelated to treatment.
Five patients had a partial response—2 in the 450 x 106 cohort and 1 in each of the other cohorts. Seven patients had a very good partial response—5 in the 450 x 106 cohort and 1 each in the 150 x 106 cohort and 800 x 106 cohort.
Updated data from this study are scheduled to be presented at the 2017 ASH Annual Meeting (abstract 740).
About breakthrough designation
The FDA’s breakthrough designation is intended to expedite the development and review of new treatments for serious or life-threatening conditions.
The designation entitles the company developing a therapy to more intensive FDA guidance on an efficient and accelerated development program, as well as eligibility for other actions to expedite FDA review, such as rolling submission and priority review.
To earn breakthrough designation, a treatment must show encouraging early clinical results demonstrating substantial improvement over available therapies with regard to a clinically significant endpoint, or it must fulfill an unmet need.
About PRIME
The EMA launched its PRIME program to enhance support for the development of medicines that target an unmet medical need.
The program involves enhanced interaction and early dialogue with developers of promising medicines to optimize development plans and speed up evaluation so these medicines can reach patients earlier.
PRIME focuses on medicines that may offer a major therapeutic advantage over existing treatments or benefit patients without treatment options. To be accepted for PRIME, a medicine must have demonstrated the potential to benefit patients with unmet medical needs based on early clinical data.
A chimeric antigen receptor (CAR) T-cell therapy, bb2121, has received breakthrough therapy designation from the US Food and Drug Administration (FDA) and was granted access to the European Medicines Agency’s (EMA’s) PRIority MEdicines (PRIME) program.
The CAR T-cell therapy is designed to target B-cell maturation antigen in previously treated patients with multiple myeloma.
bb2121 is being developed by bluebird bio, Inc., and Celgene Corporation.
The EMA’s and FDA’s decisions on bb2121 were based on preliminary data from an ongoing phase 1 study, CRB-401 (NCT02658929).
Results from this study were presented at the 2017 ASCO Annual Meeting (abstract 3010).
The study enrolled patients with relapsed and/or refractory multiple myeloma. As of the May 4, 2017 data cut-off, 21 patients had been enrolled.
Patients had a median of 7 prior lines of therapy (range, 3-14). Their previous treatments included lenalidomide and bortezomib (100%), pomalidomide and carfilzomib (91%), daratumumab (71%), and autologous stem cell transplant (100% at least once).
Twenty-nine percent of patients were refractory to bortezomib, lenalidomide, carfilzomib, pomalidomide, and daratumumab.
Patients received a conditioning regimen of cyclophosphamide and fludarabine, followed by an infusion of bb2121 at 1 of 4 doses: 50 x 106, 150 x 106, 450 x 106 and 800 x 106 CAR+ T cells.
All 21 patients were evaluable for safety.
The most common treatment-emergent grade 3-4 adverse events were cytopenias commonly associated with the lymphodepletion regimen, as well as grade 3 events of hyponatremia (n=4), upper respiratory infection (n=2), syncope (n=2), and cytokine release syndrome (CRS, n=2).
In all, 71% of patients (15/21) had CRS, mostly grade 1 and 2. For the 2 patients with grade 3 CRS, it resolved within 24 hours. To manage CRS, 4 patients received tocilizumab, and 1 (with grade 2) received steroids as well.
Eighteen patients were evaluable for efficacy, and the overall response rate was 89% (16/18).
There were 4 complete responses—2 in the 150 x 106 cohort, 1 in the 450 x 106 cohort, and 1 in the 800 x 106 cohort. The complete responder in the 450 x 106 cohort ultimately died of cardiopulmonary arrest that was deemed unrelated to treatment.
Five patients had a partial response—2 in the 450 x 106 cohort and 1 in each of the other cohorts. Seven patients had a very good partial response—5 in the 450 x 106 cohort and 1 each in the 150 x 106 cohort and 800 x 106 cohort.
Updated data from this study are scheduled to be presented at the 2017 ASH Annual Meeting (abstract 740).
About breakthrough designation
The FDA’s breakthrough designation is intended to expedite the development and review of new treatments for serious or life-threatening conditions.
The designation entitles the company developing a therapy to more intensive FDA guidance on an efficient and accelerated development program, as well as eligibility for other actions to expedite FDA review, such as rolling submission and priority review.
To earn breakthrough designation, a treatment must show encouraging early clinical results demonstrating substantial improvement over available therapies with regard to a clinically significant endpoint, or it must fulfill an unmet need.
About PRIME
The EMA launched its PRIME program to enhance support for the development of medicines that target an unmet medical need.
The program involves enhanced interaction and early dialogue with developers of promising medicines to optimize development plans and speed up evaluation so these medicines can reach patients earlier.
PRIME focuses on medicines that may offer a major therapeutic advantage over existing treatments or benefit patients without treatment options. To be accepted for PRIME, a medicine must have demonstrated the potential to benefit patients with unmet medical needs based on early clinical data.
A chimeric antigen receptor (CAR) T-cell therapy, bb2121, has received breakthrough therapy designation from the US Food and Drug Administration (FDA) and was granted access to the European Medicines Agency’s (EMA’s) PRIority MEdicines (PRIME) program.
The CAR T-cell therapy is designed to target B-cell maturation antigen in previously treated patients with multiple myeloma.
bb2121 is being developed by bluebird bio, Inc., and Celgene Corporation.
The EMA’s and FDA’s decisions on bb2121 were based on preliminary data from an ongoing phase 1 study, CRB-401 (NCT02658929).
Results from this study were presented at the 2017 ASCO Annual Meeting (abstract 3010).
The study enrolled patients with relapsed and/or refractory multiple myeloma. As of the May 4, 2017 data cut-off, 21 patients had been enrolled.
Patients had a median of 7 prior lines of therapy (range, 3-14). Their previous treatments included lenalidomide and bortezomib (100%), pomalidomide and carfilzomib (91%), daratumumab (71%), and autologous stem cell transplant (100% at least once).
Twenty-nine percent of patients were refractory to bortezomib, lenalidomide, carfilzomib, pomalidomide, and daratumumab.
Patients received a conditioning regimen of cyclophosphamide and fludarabine, followed by an infusion of bb2121 at 1 of 4 doses: 50 x 106, 150 x 106, 450 x 106 and 800 x 106 CAR+ T cells.
All 21 patients were evaluable for safety.
The most common treatment-emergent grade 3-4 adverse events were cytopenias commonly associated with the lymphodepletion regimen, as well as grade 3 events of hyponatremia (n=4), upper respiratory infection (n=2), syncope (n=2), and cytokine release syndrome (CRS, n=2).
In all, 71% of patients (15/21) had CRS, mostly grade 1 and 2. For the 2 patients with grade 3 CRS, it resolved within 24 hours. To manage CRS, 4 patients received tocilizumab, and 1 (with grade 2) received steroids as well.
Eighteen patients were evaluable for efficacy, and the overall response rate was 89% (16/18).
There were 4 complete responses—2 in the 150 x 106 cohort, 1 in the 450 x 106 cohort, and 1 in the 800 x 106 cohort. The complete responder in the 450 x 106 cohort ultimately died of cardiopulmonary arrest that was deemed unrelated to treatment.
Five patients had a partial response—2 in the 450 x 106 cohort and 1 in each of the other cohorts. Seven patients had a very good partial response—5 in the 450 x 106 cohort and 1 each in the 150 x 106 cohort and 800 x 106 cohort.
Updated data from this study are scheduled to be presented at the 2017 ASH Annual Meeting (abstract 740).
About breakthrough designation
The FDA’s breakthrough designation is intended to expedite the development and review of new treatments for serious or life-threatening conditions.
The designation entitles the company developing a therapy to more intensive FDA guidance on an efficient and accelerated development program, as well as eligibility for other actions to expedite FDA review, such as rolling submission and priority review.
To earn breakthrough designation, a treatment must show encouraging early clinical results demonstrating substantial improvement over available therapies with regard to a clinically significant endpoint, or it must fulfill an unmet need.
About PRIME
The EMA launched its PRIME program to enhance support for the development of medicines that target an unmet medical need.
The program involves enhanced interaction and early dialogue with developers of promising medicines to optimize development plans and speed up evaluation so these medicines can reach patients earlier.
PRIME focuses on medicines that may offer a major therapeutic advantage over existing treatments or benefit patients without treatment options. To be accepted for PRIME, a medicine must have demonstrated the potential to benefit patients with unmet medical needs based on early clinical data.
Antibiotics after incision and drainage for simple abscesses improves outcomes
Clinical question: Does antibiotic administration after incision and drainage of simple abscesses improve cure rates?
Background: Abscesses are the most common skin and soft-tissue infection, and most patients seek outpatient treatment. Limited data have shown that antibiotic treatment directed at Staphylococcus aureus after incision and drainage is effective, though the efficacy of adjunctive antibiotic therapy versus incision and drainage alone is unclear.
Setting: Multicenter outpatient facilities across the United States.
Synopsis: After successful incision and drainage of a simple abscess, 786 outpatients (505 adults, 281 children) were randomized to receive either clindamycin, trimethoprim-sulfamethoxazole (TMP-SMX), or placebo for 10 days. The rate of clinical cure was 83.1% in the clindamycin group, 81.7% in the TMP-SMX group, and 68.9% in the placebo group. The cure rate was significantly lower in the placebo group, compared with either of the antibiotic groups. The difference in cure rate was not significant between the clindamycin and TMP-SMX groups. Cure rates in culture-positive S. aureus patients were significantly higher in both antibiotic groups, compared with placebo. Rates of adverse events were higher in the clindamycin group (21.9%) than the TMP-SMX group (11.1%) or the placebo group (12.5%), though all adverse events resolved without sequelae.
Bottom line: Antibiotic therapy after incision and drainage for simple abscesses is associated with improved cure rate and decreased recurrence.
Citation: Daum RS, Miller LG, Immergluck L, et al. A placebo-controlled trial of antibiotics for smaller skin abscesses. N Engl J Med. 2017 Jun 29;376(26):2545-55.
Dr. Chung is hospitalist and assistant professor of medicine, Icahn School of Medicine of the Mount Sinai Health System.
Clinical question: Does antibiotic administration after incision and drainage of simple abscesses improve cure rates?
Background: Abscesses are the most common skin and soft-tissue infection, and most patients seek outpatient treatment. Limited data have shown that antibiotic treatment directed at Staphylococcus aureus after incision and drainage is effective, though the efficacy of adjunctive antibiotic therapy versus incision and drainage alone is unclear.
Setting: Multicenter outpatient facilities across the United States.
Synopsis: After successful incision and drainage of a simple abscess, 786 outpatients (505 adults, 281 children) were randomized to receive either clindamycin, trimethoprim-sulfamethoxazole (TMP-SMX), or placebo for 10 days. The rate of clinical cure was 83.1% in the clindamycin group, 81.7% in the TMP-SMX group, and 68.9% in the placebo group. The cure rate was significantly lower in the placebo group, compared with either of the antibiotic groups. The difference in cure rate was not significant between the clindamycin and TMP-SMX groups. Cure rates in culture-positive S. aureus patients were significantly higher in both antibiotic groups, compared with placebo. Rates of adverse events were higher in the clindamycin group (21.9%) than the TMP-SMX group (11.1%) or the placebo group (12.5%), though all adverse events resolved without sequelae.
Bottom line: Antibiotic therapy after incision and drainage for simple abscesses is associated with improved cure rate and decreased recurrence.
Citation: Daum RS, Miller LG, Immergluck L, et al. A placebo-controlled trial of antibiotics for smaller skin abscesses. N Engl J Med. 2017 Jun 29;376(26):2545-55.
Dr. Chung is hospitalist and assistant professor of medicine, Icahn School of Medicine of the Mount Sinai Health System.
Clinical question: Does antibiotic administration after incision and drainage of simple abscesses improve cure rates?
Background: Abscesses are the most common skin and soft-tissue infection, and most patients seek outpatient treatment. Limited data have shown that antibiotic treatment directed at Staphylococcus aureus after incision and drainage is effective, though the efficacy of adjunctive antibiotic therapy versus incision and drainage alone is unclear.
Setting: Multicenter outpatient facilities across the United States.
Synopsis: After successful incision and drainage of a simple abscess, 786 outpatients (505 adults, 281 children) were randomized to receive either clindamycin, trimethoprim-sulfamethoxazole (TMP-SMX), or placebo for 10 days. The rate of clinical cure was 83.1% in the clindamycin group, 81.7% in the TMP-SMX group, and 68.9% in the placebo group. The cure rate was significantly lower in the placebo group, compared with either of the antibiotic groups. The difference in cure rate was not significant between the clindamycin and TMP-SMX groups. Cure rates in culture-positive S. aureus patients were significantly higher in both antibiotic groups, compared with placebo. Rates of adverse events were higher in the clindamycin group (21.9%) than the TMP-SMX group (11.1%) or the placebo group (12.5%), though all adverse events resolved without sequelae.
Bottom line: Antibiotic therapy after incision and drainage for simple abscesses is associated with improved cure rate and decreased recurrence.
Citation: Daum RS, Miller LG, Immergluck L, et al. A placebo-controlled trial of antibiotics for smaller skin abscesses. N Engl J Med. 2017 Jun 29;376(26):2545-55.
Dr. Chung is hospitalist and assistant professor of medicine, Icahn School of Medicine of the Mount Sinai Health System.
From the Editors: How about that!
For all those who say that the surgeon in the trenches doesn’t have a voice in the world of organized surgery, I have a story or two for you.
About 10 or more years ago, I was at a lovely old hotel in Cooperstown, N.Y., listening to a group of elder rural surgery colleagues hold forth about feeling ignored by the American College of Surgeons and how we should all band together to form a new society of rural surgeons. Like most crowds, they were getting pretty worked up. They were pretty sure such a revolution would solve our problems and we would quit being the Rodney Dangerfields of surgery (not getting any respect, as the great comic used to say).
It took a while – a few years – for the rural surgeons and ACS leadership to get to know each other better. We did, however, get there. An Advisory Council was created with the help of some very heavy hitters not often regarded as rural champions, most of whom had roots in the rural world that helped them understand what this group of surgeons wanted and needed.
Not too much removed from this event, many surgeons were feeling isolated in general due to the many challenges of rural practice familiar to so many of us. ACS leadership sensed that surgeons needed to be connected and with considerable effort they formed listservs and found that they were successful to some degree. So, under Dr. David Hoyt’s guidance a better technology was found, resources were committed, and the ACS Communities was launched. A new era in surgeon-to-surgeon and surgeon-to-leadership communication was born. It was as if we had developed talk radio for surgeons. Some stations were loud, others came in rather softly, but all were on the air. The pulse of the Fellows became audible.
Almost simultaneously, dissatisfaction with Maintenance of Certification grew as surgeons in practice began reaching their second and third recertification and the practice of surgery became ever more specialized. Many surgeons believed that nothing would ever change, that the front-line surgeon didn’t have a chance to affect change, and that the big dogs wouldn’t listen. But the College leadership heard the voices of concern and took steps to support a new approach to certification.
So here we are in 2017, coming off a very successful Clinical Congress. The formerly obscure rural surgeon contingent had several panels on the program. Surgeons practicing in towns as small as 3,000 moderated sessions. Some of those sessions were standing room only. The College assisted (and had for a few years) those rural surgeons in organizing a Rural Surgery Dinner, which filled a restaurant full of surgeons delighted to meet in person surgeons from small communities all over the country. They shared their common experiences and planned for a better tomorrow. Later in the week, the 2nd Vice President-Elect was announced. He is a surgeon from Keokuk, IA, and one of the leaders of the rural surgeon movement within the College, Philip Caropreso, MD, FACS. A new Regent was announced: Gary Timmerman, MD, FACS. Dr. Timmerman started his career in Watertown, S.D., and now runs a rural-based surgery training program.
The Clinical Congress program included many topics and issues that Fellows from every branch of surgery had posted about on the ACS Communities. The American Board of Surgery announced sweeping coming changes to help surgeons move on from an MOC system no one was really happy with to one that has great hope of making ongoing certification more than a hoop to jump through, but instead, a real value to the Diplomate. Sitting on the main committee for the American Board of Surgery’s sprint team on certification is a surgeon from Crockett, Texas, Pat Walker, MD, FACS, who practiced surgery “on the ground” in a small community setting for three decades.
Most of us would agree that rural surgeons are facing truly daunting professional headwinds, despite their critical work in serving rural patients. Yet I have been gladdened at the response and respect that the College leadership has given to rural surgeons in recent years. The outreach by the College to support rural surgeons is part of a broader effort to hear every Fellow and make the College more relevant in the life of hard-working surgeons of every type. Changes in how we retain and improve our certification process in some significant ways came out of College efforts to listen and respond to the concerns of Fellows in the College. These and other initiatives by the College leadership show a degree of farsightedness and caring that should be gratifying to all Fellows.
When Lindsay Fox, MD, FACS, a young rural surgeon in private practice with an 8-week-old baby, receives an award for excellence, moderates her panel at the Clinical Congress to a standing-room-only crowd, and then shares her experience through the Communities, I’d say the American College of Surgeons has done an amazing job in a short period of time to make our organization reflect all of us. As Mel Allen, the voice of the Yankees in their heyday, would say, “How about that!”
Dr. Hughes is clinical professor in the department of surgery and director of medical education at the Kansas University School of Medicine, Salina Campus, and Co-Editor of ACS Surgery News.
For all those who say that the surgeon in the trenches doesn’t have a voice in the world of organized surgery, I have a story or two for you.
About 10 or more years ago, I was at a lovely old hotel in Cooperstown, N.Y., listening to a group of elder rural surgery colleagues hold forth about feeling ignored by the American College of Surgeons and how we should all band together to form a new society of rural surgeons. Like most crowds, they were getting pretty worked up. They were pretty sure such a revolution would solve our problems and we would quit being the Rodney Dangerfields of surgery (not getting any respect, as the great comic used to say).
It took a while – a few years – for the rural surgeons and ACS leadership to get to know each other better. We did, however, get there. An Advisory Council was created with the help of some very heavy hitters not often regarded as rural champions, most of whom had roots in the rural world that helped them understand what this group of surgeons wanted and needed.
Not too much removed from this event, many surgeons were feeling isolated in general due to the many challenges of rural practice familiar to so many of us. ACS leadership sensed that surgeons needed to be connected and with considerable effort they formed listservs and found that they were successful to some degree. So, under Dr. David Hoyt’s guidance a better technology was found, resources were committed, and the ACS Communities was launched. A new era in surgeon-to-surgeon and surgeon-to-leadership communication was born. It was as if we had developed talk radio for surgeons. Some stations were loud, others came in rather softly, but all were on the air. The pulse of the Fellows became audible.
Almost simultaneously, dissatisfaction with Maintenance of Certification grew as surgeons in practice began reaching their second and third recertification and the practice of surgery became ever more specialized. Many surgeons believed that nothing would ever change, that the front-line surgeon didn’t have a chance to affect change, and that the big dogs wouldn’t listen. But the College leadership heard the voices of concern and took steps to support a new approach to certification.
So here we are in 2017, coming off a very successful Clinical Congress. The formerly obscure rural surgeon contingent had several panels on the program. Surgeons practicing in towns as small as 3,000 moderated sessions. Some of those sessions were standing room only. The College assisted (and had for a few years) those rural surgeons in organizing a Rural Surgery Dinner, which filled a restaurant full of surgeons delighted to meet in person surgeons from small communities all over the country. They shared their common experiences and planned for a better tomorrow. Later in the week, the 2nd Vice President-Elect was announced. He is a surgeon from Keokuk, IA, and one of the leaders of the rural surgeon movement within the College, Philip Caropreso, MD, FACS. A new Regent was announced: Gary Timmerman, MD, FACS. Dr. Timmerman started his career in Watertown, S.D., and now runs a rural-based surgery training program.
The Clinical Congress program included many topics and issues that Fellows from every branch of surgery had posted about on the ACS Communities. The American Board of Surgery announced sweeping coming changes to help surgeons move on from an MOC system no one was really happy with to one that has great hope of making ongoing certification more than a hoop to jump through, but instead, a real value to the Diplomate. Sitting on the main committee for the American Board of Surgery’s sprint team on certification is a surgeon from Crockett, Texas, Pat Walker, MD, FACS, who practiced surgery “on the ground” in a small community setting for three decades.
Most of us would agree that rural surgeons are facing truly daunting professional headwinds, despite their critical work in serving rural patients. Yet I have been gladdened at the response and respect that the College leadership has given to rural surgeons in recent years. The outreach by the College to support rural surgeons is part of a broader effort to hear every Fellow and make the College more relevant in the life of hard-working surgeons of every type. Changes in how we retain and improve our certification process in some significant ways came out of College efforts to listen and respond to the concerns of Fellows in the College. These and other initiatives by the College leadership show a degree of farsightedness and caring that should be gratifying to all Fellows.
When Lindsay Fox, MD, FACS, a young rural surgeon in private practice with an 8-week-old baby, receives an award for excellence, moderates her panel at the Clinical Congress to a standing-room-only crowd, and then shares her experience through the Communities, I’d say the American College of Surgeons has done an amazing job in a short period of time to make our organization reflect all of us. As Mel Allen, the voice of the Yankees in their heyday, would say, “How about that!”
Dr. Hughes is clinical professor in the department of surgery and director of medical education at the Kansas University School of Medicine, Salina Campus, and Co-Editor of ACS Surgery News.
For all those who say that the surgeon in the trenches doesn’t have a voice in the world of organized surgery, I have a story or two for you.
About 10 or more years ago, I was at a lovely old hotel in Cooperstown, N.Y., listening to a group of elder rural surgery colleagues hold forth about feeling ignored by the American College of Surgeons and how we should all band together to form a new society of rural surgeons. Like most crowds, they were getting pretty worked up. They were pretty sure such a revolution would solve our problems and we would quit being the Rodney Dangerfields of surgery (not getting any respect, as the great comic used to say).
It took a while – a few years – for the rural surgeons and ACS leadership to get to know each other better. We did, however, get there. An Advisory Council was created with the help of some very heavy hitters not often regarded as rural champions, most of whom had roots in the rural world that helped them understand what this group of surgeons wanted and needed.
Not too much removed from this event, many surgeons were feeling isolated in general due to the many challenges of rural practice familiar to so many of us. ACS leadership sensed that surgeons needed to be connected and with considerable effort they formed listservs and found that they were successful to some degree. So, under Dr. David Hoyt’s guidance a better technology was found, resources were committed, and the ACS Communities was launched. A new era in surgeon-to-surgeon and surgeon-to-leadership communication was born. It was as if we had developed talk radio for surgeons. Some stations were loud, others came in rather softly, but all were on the air. The pulse of the Fellows became audible.
Almost simultaneously, dissatisfaction with Maintenance of Certification grew as surgeons in practice began reaching their second and third recertification and the practice of surgery became ever more specialized. Many surgeons believed that nothing would ever change, that the front-line surgeon didn’t have a chance to affect change, and that the big dogs wouldn’t listen. But the College leadership heard the voices of concern and took steps to support a new approach to certification.
So here we are in 2017, coming off a very successful Clinical Congress. The formerly obscure rural surgeon contingent had several panels on the program. Surgeons practicing in towns as small as 3,000 moderated sessions. Some of those sessions were standing room only. The College assisted (and had for a few years) those rural surgeons in organizing a Rural Surgery Dinner, which filled a restaurant full of surgeons delighted to meet in person surgeons from small communities all over the country. They shared their common experiences and planned for a better tomorrow. Later in the week, the 2nd Vice President-Elect was announced. He is a surgeon from Keokuk, IA, and one of the leaders of the rural surgeon movement within the College, Philip Caropreso, MD, FACS. A new Regent was announced: Gary Timmerman, MD, FACS. Dr. Timmerman started his career in Watertown, S.D., and now runs a rural-based surgery training program.
The Clinical Congress program included many topics and issues that Fellows from every branch of surgery had posted about on the ACS Communities. The American Board of Surgery announced sweeping coming changes to help surgeons move on from an MOC system no one was really happy with to one that has great hope of making ongoing certification more than a hoop to jump through, but instead, a real value to the Diplomate. Sitting on the main committee for the American Board of Surgery’s sprint team on certification is a surgeon from Crockett, Texas, Pat Walker, MD, FACS, who practiced surgery “on the ground” in a small community setting for three decades.
Most of us would agree that rural surgeons are facing truly daunting professional headwinds, despite their critical work in serving rural patients. Yet I have been gladdened at the response and respect that the College leadership has given to rural surgeons in recent years. The outreach by the College to support rural surgeons is part of a broader effort to hear every Fellow and make the College more relevant in the life of hard-working surgeons of every type. Changes in how we retain and improve our certification process in some significant ways came out of College efforts to listen and respond to the concerns of Fellows in the College. These and other initiatives by the College leadership show a degree of farsightedness and caring that should be gratifying to all Fellows.
When Lindsay Fox, MD, FACS, a young rural surgeon in private practice with an 8-week-old baby, receives an award for excellence, moderates her panel at the Clinical Congress to a standing-room-only crowd, and then shares her experience through the Communities, I’d say the American College of Surgeons has done an amazing job in a short period of time to make our organization reflect all of us. As Mel Allen, the voice of the Yankees in their heyday, would say, “How about that!”
Dr. Hughes is clinical professor in the department of surgery and director of medical education at the Kansas University School of Medicine, Salina Campus, and Co-Editor of ACS Surgery News.
Benralizumab approved for eosinophilic asthma
This is the only respiratory biologic to provide fast and “near-complete depletion of eosinophils within 24 hours,” according to the statement from AstraZeneca.
Similar adverse events were seen in patients who took benralizumab and the placebo. AstraZeneca will market benralizumab under the trade name Fasenra.
“This is an important day for severe, eosinophilic asthma patients who have had limited treatment options for far too long,” said Eugene Bleecker, MD, professor and codirector of genetics, genomics, and precision medicine at the University of Arizona in Tucson, in the statement.
This is the only respiratory biologic to provide fast and “near-complete depletion of eosinophils within 24 hours,” according to the statement from AstraZeneca.
Similar adverse events were seen in patients who took benralizumab and the placebo. AstraZeneca will market benralizumab under the trade name Fasenra.
“This is an important day for severe, eosinophilic asthma patients who have had limited treatment options for far too long,” said Eugene Bleecker, MD, professor and codirector of genetics, genomics, and precision medicine at the University of Arizona in Tucson, in the statement.
This is the only respiratory biologic to provide fast and “near-complete depletion of eosinophils within 24 hours,” according to the statement from AstraZeneca.
Similar adverse events were seen in patients who took benralizumab and the placebo. AstraZeneca will market benralizumab under the trade name Fasenra.
“This is an important day for severe, eosinophilic asthma patients who have had limited treatment options for far too long,” said Eugene Bleecker, MD, professor and codirector of genetics, genomics, and precision medicine at the University of Arizona in Tucson, in the statement.
FDA addresses cell-based regenerative medicine in comprehensive new policy
The Food and Drug Administration has announced a new policy that addresses the rapid growth and development of regenerative medicine products, which include novel cellular therapies, with the aim of ensuring their safety and effectiveness.
“The framework – outlined in a suite of four guidance documents – builds upon the FDA’s existing risk-based regulatory approach to more clearly describe what products are regulated as drugs, devices, and/or biological products,” the FDA announced in a statement released on Nov. 16.
He added: “This is no longer the stuff of science fiction. This is the practical promise of modern applications of regenerative medicine.” But, while advances have benefited many patients, he referred to a small number of “unscrupulous actors” that have provided treatments that have harmed patients, which is why stricter FDA enforcement is needed.
Clarification of the existing regulations will “promote responsible and flexible regulation that leverages science to advance public health,” Dr. Gottlieb said during a media briefing held by the FDA to discuss the new framework.
During the briefing, in response to a question concerning adipose tissue injections and their associated risks, Peter Marks, MD, director of the FDA’s Center for Biologics Evaluation and Research, explained that the guidance documents will clearly delineate when adipose tissue will be classified as a structural tissue and a stem cell product. If a provider is purveying dangerous products, the FDA can “when necessary, undertake seizures, ask for injunctions, and in some cases when it has been determined that certain violations have occurred even criminal actions can be taken,” he said.
Earlier this year, a report of three patients who had severe visual loss after treatment with intravitreal injections of autologous adipose tissue for age-related macular degeneration was published (N Engl J Med. 2017;376:1047-53).
The new framework is composed of two final guidance documents and two draft guidance documents. The first final guidance document provides details about regulations concerning cell and tissue-based products and when those products are subject to regulation in surgical procedures. The second final guidance document elaborates on the definition of “minimal manipulation” and “homologous use” with the hopes of clarifying what products are subject to regulation. These documents will also explain how the FDA will provide a framework for premarket authorization for cell-based regenerative products.
One draft guidance outlines the FDA’s plan to simplify and expedite the application of the regulatory requirements for devices used in relation to regenerative medicine advanced therapies; the second draft guidance outlines the expedited programs that may be available to sponsors of regenerative therapies.
The Food and Drug Administration has announced a new policy that addresses the rapid growth and development of regenerative medicine products, which include novel cellular therapies, with the aim of ensuring their safety and effectiveness.
“The framework – outlined in a suite of four guidance documents – builds upon the FDA’s existing risk-based regulatory approach to more clearly describe what products are regulated as drugs, devices, and/or biological products,” the FDA announced in a statement released on Nov. 16.
He added: “This is no longer the stuff of science fiction. This is the practical promise of modern applications of regenerative medicine.” But, while advances have benefited many patients, he referred to a small number of “unscrupulous actors” that have provided treatments that have harmed patients, which is why stricter FDA enforcement is needed.
Clarification of the existing regulations will “promote responsible and flexible regulation that leverages science to advance public health,” Dr. Gottlieb said during a media briefing held by the FDA to discuss the new framework.
During the briefing, in response to a question concerning adipose tissue injections and their associated risks, Peter Marks, MD, director of the FDA’s Center for Biologics Evaluation and Research, explained that the guidance documents will clearly delineate when adipose tissue will be classified as a structural tissue and a stem cell product. If a provider is purveying dangerous products, the FDA can “when necessary, undertake seizures, ask for injunctions, and in some cases when it has been determined that certain violations have occurred even criminal actions can be taken,” he said.
Earlier this year, a report of three patients who had severe visual loss after treatment with intravitreal injections of autologous adipose tissue for age-related macular degeneration was published (N Engl J Med. 2017;376:1047-53).
The new framework is composed of two final guidance documents and two draft guidance documents. The first final guidance document provides details about regulations concerning cell and tissue-based products and when those products are subject to regulation in surgical procedures. The second final guidance document elaborates on the definition of “minimal manipulation” and “homologous use” with the hopes of clarifying what products are subject to regulation. These documents will also explain how the FDA will provide a framework for premarket authorization for cell-based regenerative products.
One draft guidance outlines the FDA’s plan to simplify and expedite the application of the regulatory requirements for devices used in relation to regenerative medicine advanced therapies; the second draft guidance outlines the expedited programs that may be available to sponsors of regenerative therapies.
The Food and Drug Administration has announced a new policy that addresses the rapid growth and development of regenerative medicine products, which include novel cellular therapies, with the aim of ensuring their safety and effectiveness.
“The framework – outlined in a suite of four guidance documents – builds upon the FDA’s existing risk-based regulatory approach to more clearly describe what products are regulated as drugs, devices, and/or biological products,” the FDA announced in a statement released on Nov. 16.
He added: “This is no longer the stuff of science fiction. This is the practical promise of modern applications of regenerative medicine.” But, while advances have benefited many patients, he referred to a small number of “unscrupulous actors” that have provided treatments that have harmed patients, which is why stricter FDA enforcement is needed.
Clarification of the existing regulations will “promote responsible and flexible regulation that leverages science to advance public health,” Dr. Gottlieb said during a media briefing held by the FDA to discuss the new framework.
During the briefing, in response to a question concerning adipose tissue injections and their associated risks, Peter Marks, MD, director of the FDA’s Center for Biologics Evaluation and Research, explained that the guidance documents will clearly delineate when adipose tissue will be classified as a structural tissue and a stem cell product. If a provider is purveying dangerous products, the FDA can “when necessary, undertake seizures, ask for injunctions, and in some cases when it has been determined that certain violations have occurred even criminal actions can be taken,” he said.
Earlier this year, a report of three patients who had severe visual loss after treatment with intravitreal injections of autologous adipose tissue for age-related macular degeneration was published (N Engl J Med. 2017;376:1047-53).
The new framework is composed of two final guidance documents and two draft guidance documents. The first final guidance document provides details about regulations concerning cell and tissue-based products and when those products are subject to regulation in surgical procedures. The second final guidance document elaborates on the definition of “minimal manipulation” and “homologous use” with the hopes of clarifying what products are subject to regulation. These documents will also explain how the FDA will provide a framework for premarket authorization for cell-based regenerative products.
One draft guidance outlines the FDA’s plan to simplify and expedite the application of the regulatory requirements for devices used in relation to regenerative medicine advanced therapies; the second draft guidance outlines the expedited programs that may be available to sponsors of regenerative therapies.
FROM an FDA MEDIA BRIEFING
Career (of your dreams) advice for young MIGS surgeons
From the 46th AAGL Global Congress on MIGS
From the 46th AAGL Global Congress on MIGS
From the 46th AAGL Global Congress on MIGS
AGA launches new registry to track patient outcomes
The AGA Center for GI Innovation and Technology is excited to announce a new clinical research registry to track and evaluate patient outcomes after trans-oral endoscopic suturing procedures.
The Prospective Registry for Trans-Oral Suturing Applications (“Endoscopic Suturing Registry”) will collect real-world data related to the safety and effectiveness of procedures done with Apollo Endosurgery’s OverStitch™ Endoscopic Suturing System. Jennifer Maranki, MD, director of endoscopy, Penn State Milton S. Hershey School of Medicine, and Brian Dunkin, MD, head of endoscopic surgery and medical director, Houston Methodist Institute for Technology, Innovation and Education, will serve as principal investigators for the Endoscopic Suturing Registry. The Registry will begin collecting patient data in early 2018.
We asked Michael Kochman, MD, AGAF, past chair of the AGA Center for GI Innovation and Technology and director of the Center for Endoscopic Innovation, Research and Training at the University of Pennsylvania, to weigh in on the value of this new registry.
“Flexible endoscopic suturing is an important tool for the treatment of a number of GI disorders. As these procedures become more routine in GI and surgery practices across the country, the real-world data AGA will collect through the Endoscopic Suturing Registry will guide all stakeholders in making informed decisions around the continued adoption of these procedures in clinical practice.”
Learn more about AGA’s registry initiative at www.gastro.org/patient-care/registries-studies.
The AGA Center for GI Innovation and Technology is excited to announce a new clinical research registry to track and evaluate patient outcomes after trans-oral endoscopic suturing procedures.
The Prospective Registry for Trans-Oral Suturing Applications (“Endoscopic Suturing Registry”) will collect real-world data related to the safety and effectiveness of procedures done with Apollo Endosurgery’s OverStitch™ Endoscopic Suturing System. Jennifer Maranki, MD, director of endoscopy, Penn State Milton S. Hershey School of Medicine, and Brian Dunkin, MD, head of endoscopic surgery and medical director, Houston Methodist Institute for Technology, Innovation and Education, will serve as principal investigators for the Endoscopic Suturing Registry. The Registry will begin collecting patient data in early 2018.
We asked Michael Kochman, MD, AGAF, past chair of the AGA Center for GI Innovation and Technology and director of the Center for Endoscopic Innovation, Research and Training at the University of Pennsylvania, to weigh in on the value of this new registry.
“Flexible endoscopic suturing is an important tool for the treatment of a number of GI disorders. As these procedures become more routine in GI and surgery practices across the country, the real-world data AGA will collect through the Endoscopic Suturing Registry will guide all stakeholders in making informed decisions around the continued adoption of these procedures in clinical practice.”
Learn more about AGA’s registry initiative at www.gastro.org/patient-care/registries-studies.
The AGA Center for GI Innovation and Technology is excited to announce a new clinical research registry to track and evaluate patient outcomes after trans-oral endoscopic suturing procedures.
The Prospective Registry for Trans-Oral Suturing Applications (“Endoscopic Suturing Registry”) will collect real-world data related to the safety and effectiveness of procedures done with Apollo Endosurgery’s OverStitch™ Endoscopic Suturing System. Jennifer Maranki, MD, director of endoscopy, Penn State Milton S. Hershey School of Medicine, and Brian Dunkin, MD, head of endoscopic surgery and medical director, Houston Methodist Institute for Technology, Innovation and Education, will serve as principal investigators for the Endoscopic Suturing Registry. The Registry will begin collecting patient data in early 2018.
We asked Michael Kochman, MD, AGAF, past chair of the AGA Center for GI Innovation and Technology and director of the Center for Endoscopic Innovation, Research and Training at the University of Pennsylvania, to weigh in on the value of this new registry.
“Flexible endoscopic suturing is an important tool for the treatment of a number of GI disorders. As these procedures become more routine in GI and surgery practices across the country, the real-world data AGA will collect through the Endoscopic Suturing Registry will guide all stakeholders in making informed decisions around the continued adoption of these procedures in clinical practice.”
Learn more about AGA’s registry initiative at www.gastro.org/patient-care/registries-studies.
PCVs may reduce AOM severity, although they do not decrease incidence
Use of the 7-valent pneumococcal conjugate vaccine (PCV7) may have had a part in reducing the incidence of severe middle ear inflammation of acute otitis media (AOM) in Japanese children, decreasing the rate of myringotomies for AOM, said Atsushi Sasaki, of the Hiroshima (Japan) University, and associates.
To assess whether use of PCV7 and then PCV13 affected AOM incidence, the investigators looked at the incidence of visits to medical institutions (VtMI) due to all-cause AOM in children younger than 15 years in the Japan Medical Data Center Claims Database between January 2005 and December 2015. Data for children aged 10 years to younger than 15 years served as the control. The rate of myringotomies for AOM (MyfA) from January 2007 to December 2015 also was assessed.
Numerous other studies have “concluded that the preventive effect of PCV7 against AOM was very modest,” the researchers said. “In contrast, our study proposes the hypothesis that PCV7 use in 1-year-olds may contribute to the decreased incidence of severe middle ear inflammation of AOM. When evaluating the effectiveness of PCV, measures to evaluate severity may be as important as evaluating disease prevention.”
Read more in Auris Nasus Larynx (2017 Nov 1. doi: 10.1016/j.anl.2017.10.006).
Use of the 7-valent pneumococcal conjugate vaccine (PCV7) may have had a part in reducing the incidence of severe middle ear inflammation of acute otitis media (AOM) in Japanese children, decreasing the rate of myringotomies for AOM, said Atsushi Sasaki, of the Hiroshima (Japan) University, and associates.
To assess whether use of PCV7 and then PCV13 affected AOM incidence, the investigators looked at the incidence of visits to medical institutions (VtMI) due to all-cause AOM in children younger than 15 years in the Japan Medical Data Center Claims Database between January 2005 and December 2015. Data for children aged 10 years to younger than 15 years served as the control. The rate of myringotomies for AOM (MyfA) from January 2007 to December 2015 also was assessed.
Numerous other studies have “concluded that the preventive effect of PCV7 against AOM was very modest,” the researchers said. “In contrast, our study proposes the hypothesis that PCV7 use in 1-year-olds may contribute to the decreased incidence of severe middle ear inflammation of AOM. When evaluating the effectiveness of PCV, measures to evaluate severity may be as important as evaluating disease prevention.”
Read more in Auris Nasus Larynx (2017 Nov 1. doi: 10.1016/j.anl.2017.10.006).
Use of the 7-valent pneumococcal conjugate vaccine (PCV7) may have had a part in reducing the incidence of severe middle ear inflammation of acute otitis media (AOM) in Japanese children, decreasing the rate of myringotomies for AOM, said Atsushi Sasaki, of the Hiroshima (Japan) University, and associates.
To assess whether use of PCV7 and then PCV13 affected AOM incidence, the investigators looked at the incidence of visits to medical institutions (VtMI) due to all-cause AOM in children younger than 15 years in the Japan Medical Data Center Claims Database between January 2005 and December 2015. Data for children aged 10 years to younger than 15 years served as the control. The rate of myringotomies for AOM (MyfA) from January 2007 to December 2015 also was assessed.
Numerous other studies have “concluded that the preventive effect of PCV7 against AOM was very modest,” the researchers said. “In contrast, our study proposes the hypothesis that PCV7 use in 1-year-olds may contribute to the decreased incidence of severe middle ear inflammation of AOM. When evaluating the effectiveness of PCV, measures to evaluate severity may be as important as evaluating disease prevention.”
Read more in Auris Nasus Larynx (2017 Nov 1. doi: 10.1016/j.anl.2017.10.006).
FROM AURIS NASUS LARYNX