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The US Food and Drug Administration (FDA) has granted accelerated approval for panobinostat (Farydak) to treat patients with multiple myeloma (MM).
Panobinostat is the first histone deacetylase (HDAC) inhibitor approved to treat MM.
The drug can now be used in combination with bortezomib and dexamethasone to treat patients who have received at least 2 prior standard therapies, including bortezomib and an immunomodulatory agent (IMiD).
Panobinostat was approved with a boxed warning alerting patients and healthcare professionals that severe diarrhea and severe and fatal cardiac events, arrhythmias, and electrocardiogram changes have occurred in patients receiving the drug.
Panobinostat was approved with a Risk Evaluation and Mitigation Strategy as well, which consists of a communication plan to inform healthcare professionals of these risks and how to minimize them.
Data supporting approval
In November 2014, the FDA’s Oncologic Drugs Advisory Committee advised the agency that, based on the data reviewed, the benefits of panobinostat did not outweigh its risks for patients with relapsed MM.
After the meeting, Novartis, the company developing the HDAC inhibitor, submitted additional information supporting the use of panobinostat for a different indication: MM patients who have received at least 2 prior standard therapies, including bortezomib and an IMiD.
The FDA’s accelerated approval of panobinostat is based on that data—efficacy and safety results in a subgroup analysis of 193 patients enrolled in the phase 3 PANORAMA-1 trial. These patients had received prior treatment with both bortezomib and an IMiD.
In these patients, treatment with panobinostat, bortezomib, and dexamethasone resulted in superior progression-free survival, when compared to treatment with bortezomib, dexamethasone, and placebo—10.6 months and 5.8 months, respectively (hazard ratio=0.52).
The most common adverse events (incidence ≥ 20%) in clinical studies of panobinostat have been diarrhea, fatigue, nausea, peripheral edema, decreased appetite, pyrexia, and vomiting.
The most common non-hematologic laboratory abnormalities (incidence ≥ 40%) were hypophosphatemia, hypokalemia, hyponatremia, and increased creatinine. The most common hematologic laboratory abnormalities (incidence ≥ 60%) were thrombocytopenia, lymphopenia, leukopenia, neutropenia, and anemia.
Panobinostat can cause fatal and serious toxicities, including severe diarrhea and cardiac toxicities.
The most frequent (≥ 5%) treatment-emergent serious adverse events for patients treated with the HDAC inhibitor were pneumonia (18%), diarrhea (11%), thrombocytopenia (7%), fatigue (6%), and sepsis (6%). Additional serious adverse events included hemorrhage, myelosuppression, infections, hepatotoxicity, and embryo-fetal toxicity.
Panobinostat development
The FDA previously granted panobinostat priority review and orphan product designation. Priority review provides an expedited review of drugs that are intended to treat a serious disease or condition and may provide a significant improvement over available therapy. Orphan product designation is given to drugs intended to treat rare diseases.
Now, the FDA has granted panobinostat accelerated approval, which allows for conditional approval of a drug based on clinical data showing the drug has an effect on a surrogate endpoint reasonably likely to predict clinical benefit to patients.
Continued approval of panobinostat may be contingent upon verification of a clinical benefit in confirmatory trials conducted by Novartis. An improvement in overall survival or disease-related symptoms has not yet been established for the HDAC inhibitor.
For more details on panobinostat, see the full prescribing information. 
The US Food and Drug Administration (FDA) has granted accelerated approval for panobinostat (Farydak) to treat patients with multiple myeloma (MM).
Panobinostat is the first histone deacetylase (HDAC) inhibitor approved to treat MM.
The drug can now be used in combination with bortezomib and dexamethasone to treat patients who have received at least 2 prior standard therapies, including bortezomib and an immunomodulatory agent (IMiD).
Panobinostat was approved with a boxed warning alerting patients and healthcare professionals that severe diarrhea and severe and fatal cardiac events, arrhythmias, and electrocardiogram changes have occurred in patients receiving the drug.
Panobinostat was approved with a Risk Evaluation and Mitigation Strategy as well, which consists of a communication plan to inform healthcare professionals of these risks and how to minimize them.
Data supporting approval
In November 2014, the FDA’s Oncologic Drugs Advisory Committee advised the agency that, based on the data reviewed, the benefits of panobinostat did not outweigh its risks for patients with relapsed MM.
After the meeting, Novartis, the company developing the HDAC inhibitor, submitted additional information supporting the use of panobinostat for a different indication: MM patients who have received at least 2 prior standard therapies, including bortezomib and an IMiD.
The FDA’s accelerated approval of panobinostat is based on that data—efficacy and safety results in a subgroup analysis of 193 patients enrolled in the phase 3 PANORAMA-1 trial. These patients had received prior treatment with both bortezomib and an IMiD.
In these patients, treatment with panobinostat, bortezomib, and dexamethasone resulted in superior progression-free survival, when compared to treatment with bortezomib, dexamethasone, and placebo—10.6 months and 5.8 months, respectively (hazard ratio=0.52).
The most common adverse events (incidence ≥ 20%) in clinical studies of panobinostat have been diarrhea, fatigue, nausea, peripheral edema, decreased appetite, pyrexia, and vomiting.
The most common non-hematologic laboratory abnormalities (incidence ≥ 40%) were hypophosphatemia, hypokalemia, hyponatremia, and increased creatinine. The most common hematologic laboratory abnormalities (incidence ≥ 60%) were thrombocytopenia, lymphopenia, leukopenia, neutropenia, and anemia.
Panobinostat can cause fatal and serious toxicities, including severe diarrhea and cardiac toxicities.
The most frequent (≥ 5%) treatment-emergent serious adverse events for patients treated with the HDAC inhibitor were pneumonia (18%), diarrhea (11%), thrombocytopenia (7%), fatigue (6%), and sepsis (6%). Additional serious adverse events included hemorrhage, myelosuppression, infections, hepatotoxicity, and embryo-fetal toxicity.
Panobinostat development
The FDA previously granted panobinostat priority review and orphan product designation. Priority review provides an expedited review of drugs that are intended to treat a serious disease or condition and may provide a significant improvement over available therapy. Orphan product designation is given to drugs intended to treat rare diseases.
Now, the FDA has granted panobinostat accelerated approval, which allows for conditional approval of a drug based on clinical data showing the drug has an effect on a surrogate endpoint reasonably likely to predict clinical benefit to patients.
Continued approval of panobinostat may be contingent upon verification of a clinical benefit in confirmatory trials conducted by Novartis. An improvement in overall survival or disease-related symptoms has not yet been established for the HDAC inhibitor.
For more details on panobinostat, see the full prescribing information.
The US Food and Drug Administration (FDA) has granted accelerated approval for panobinostat (Farydak) to treat patients with multiple myeloma (MM).
Panobinostat is the first histone deacetylase (HDAC) inhibitor approved to treat MM.
The drug can now be used in combination with bortezomib and dexamethasone to treat patients who have received at least 2 prior standard therapies, including bortezomib and an immunomodulatory agent (IMiD).
Panobinostat was approved with a boxed warning alerting patients and healthcare professionals that severe diarrhea and severe and fatal cardiac events, arrhythmias, and electrocardiogram changes have occurred in patients receiving the drug.
Panobinostat was approved with a Risk Evaluation and Mitigation Strategy as well, which consists of a communication plan to inform healthcare professionals of these risks and how to minimize them.
Data supporting approval
In November 2014, the FDA’s Oncologic Drugs Advisory Committee advised the agency that, based on the data reviewed, the benefits of panobinostat did not outweigh its risks for patients with relapsed MM.
After the meeting, Novartis, the company developing the HDAC inhibitor, submitted additional information supporting the use of panobinostat for a different indication: MM patients who have received at least 2 prior standard therapies, including bortezomib and an IMiD.
The FDA’s accelerated approval of panobinostat is based on that data—efficacy and safety results in a subgroup analysis of 193 patients enrolled in the phase 3 PANORAMA-1 trial. These patients had received prior treatment with both bortezomib and an IMiD.
In these patients, treatment with panobinostat, bortezomib, and dexamethasone resulted in superior progression-free survival, when compared to treatment with bortezomib, dexamethasone, and placebo—10.6 months and 5.8 months, respectively (hazard ratio=0.52).
The most common adverse events (incidence ≥ 20%) in clinical studies of panobinostat have been diarrhea, fatigue, nausea, peripheral edema, decreased appetite, pyrexia, and vomiting.
The most common non-hematologic laboratory abnormalities (incidence ≥ 40%) were hypophosphatemia, hypokalemia, hyponatremia, and increased creatinine. The most common hematologic laboratory abnormalities (incidence ≥ 60%) were thrombocytopenia, lymphopenia, leukopenia, neutropenia, and anemia.
Panobinostat can cause fatal and serious toxicities, including severe diarrhea and cardiac toxicities.
The most frequent (≥ 5%) treatment-emergent serious adverse events for patients treated with the HDAC inhibitor were pneumonia (18%), diarrhea (11%), thrombocytopenia (7%), fatigue (6%), and sepsis (6%). Additional serious adverse events included hemorrhage, myelosuppression, infections, hepatotoxicity, and embryo-fetal toxicity.
Panobinostat development
The FDA previously granted panobinostat priority review and orphan product designation. Priority review provides an expedited review of drugs that are intended to treat a serious disease or condition and may provide a significant improvement over available therapy. Orphan product designation is given to drugs intended to treat rare diseases.
Now, the FDA has granted panobinostat accelerated approval, which allows for conditional approval of a drug based on clinical data showing the drug has an effect on a surrogate endpoint reasonably likely to predict clinical benefit to patients.
Continued approval of panobinostat may be contingent upon verification of a clinical benefit in confirmatory trials conducted by Novartis. An improvement in overall survival or disease-related symptoms has not yet been established for the HDAC inhibitor.
For more details on panobinostat, see the full prescribing information.