FDA approves new drug for hemophilia B

Antihemophilic factor

The US Food and Drug Administration (FDA) has approved an intravenous, recombinant, human coagulation factor IX product (Ixinity) for use in patients

with hemophilia B.

The drug is intended to control and prevent bleeding episodes and for perioperative management in adults and children age 12 and older.

Concurrent with the FDA’s approval, Emergent Biosolutions (the company developing Ixinity) launched the Ixinity IXperience Concierge. This resource, which consumers can access by calling 1-855-IXINITY, provides information on the drug.

About Ixinity

Ixinity contains trenonacog alfa, a purified, single-chain glycoprotein derived from Chinese hamster ovary (CHO) cells that has an amino acid sequence comparable to the Thr148 allelic form of plasma-derived factor IX.

No human or animal proteins are added during any stage of manufacturing or formulation of Ixinity. The recombinant factor IX is purified by a chromatography purification process.

The process includes 3 validated steps for virus inactivation and removal. It also includes a validated manufacturing step to reduce the presence of CHO proteins in the final drug product.

Ixinity is contraindicated in patients who have known hypersensitivity to the drug or its excipients, including CHO protein. Hypersensitivity reactions, including anaphylaxis, may occur following treatment with Ixinity. Patients who receive Ixinity are also at risk of developing nephrotic syndrome and thromboembolism.

Trial data

The FDA approved Ixinity based on results from a phase 1/3 trial of the drug in previously treated adults and children (age 12 and older) with severe to moderately severe (factor IX level < 2%) hemophilia B.

Seventy-seven patients received at least 1 dose of Ixinity. The drug was given as routine prophylaxis or on-demand treatment for bleeding episodes.

Fifty-five patients received treatment for more than 50 exposure days, and 45 received the drug for more than 100 exposure days. The median duration of treatment on study was 16.2 months (range, 2.4-39.6 months) for the routine treatment regimen and 14.1 months (range, 2.3-36.9 months) for the on-demand treatment regimen.

A total of 508 bleeding episodes were treated with Ixinity—286 bleeds for patients on routine treatment and 222 for patients receiving on-demand treatment. A majority of the bleeds (84%) were resolved by 1 or 2 infusions of the drug.

Patients rated hemostatic efficacy at the resolution of a bleed as “excellent” or “good” in 84% of all treated bleeding episodes. “Excellent” was defined as a dramatic response with abrupt pain relief and clear reduction in joint or hemorrhage site size. “Good” was defined as pain relief or reduction in hemorrhage site size that may have required an additional infusion for resolution.

Ixinity also induced hemostasis in patients who underwent major surgical procedures.

The drug exhibited similar pharmacokinetic behavior as nonacog alfa, another licensed recombinant coagulation factor IX product. There was no significant reduction in steady-state factor IX levels or alteration in pharmacokinetic behavior over time with Ixinity.

There were 14 adverse events reported in 6 patients. The most common event, observed in 2.6% of patients, was headache. Other adverse events were asthenia, apathy, depression, dysgeusia, influenza, injection site discomfort, lethargy, and skin rash.

None of the patients developed inhibitors to Ixinity, and there were no reports of thrombotic events or allergic reactions.

For more details on this research, see the full prescribing information for Ixinity, available at www.IXINITY.com.

Antihemophilic factor

The US Food and Drug Administration (FDA) has approved an intravenous, recombinant, human coagulation factor IX product (Ixinity) for use in patients

with hemophilia B.

The drug is intended to control and prevent bleeding episodes and for perioperative management in adults and children age 12 and older.

Concurrent with the FDA’s approval, Emergent Biosolutions (the company developing Ixinity) launched the Ixinity IXperience Concierge. This resource, which consumers can access by calling 1-855-IXINITY, provides information on the drug.

About Ixinity

Ixinity contains trenonacog alfa, a purified, single-chain glycoprotein derived from Chinese hamster ovary (CHO) cells that has an amino acid sequence comparable to the Thr148 allelic form of plasma-derived factor IX.

No human or animal proteins are added during any stage of manufacturing or formulation of Ixinity. The recombinant factor IX is purified by a chromatography purification process.

The process includes 3 validated steps for virus inactivation and removal. It also includes a validated manufacturing step to reduce the presence of CHO proteins in the final drug product.

Ixinity is contraindicated in patients who have known hypersensitivity to the drug or its excipients, including CHO protein. Hypersensitivity reactions, including anaphylaxis, may occur following treatment with Ixinity. Patients who receive Ixinity are also at risk of developing nephrotic syndrome and thromboembolism.

Trial data

The FDA approved Ixinity based on results from a phase 1/3 trial of the drug in previously treated adults and children (age 12 and older) with severe to moderately severe (factor IX level < 2%) hemophilia B.

Seventy-seven patients received at least 1 dose of Ixinity. The drug was given as routine prophylaxis or on-demand treatment for bleeding episodes.

Fifty-five patients received treatment for more than 50 exposure days, and 45 received the drug for more than 100 exposure days. The median duration of treatment on study was 16.2 months (range, 2.4-39.6 months) for the routine treatment regimen and 14.1 months (range, 2.3-36.9 months) for the on-demand treatment regimen.

A total of 508 bleeding episodes were treated with Ixinity—286 bleeds for patients on routine treatment and 222 for patients receiving on-demand treatment. A majority of the bleeds (84%) were resolved by 1 or 2 infusions of the drug.

Patients rated hemostatic efficacy at the resolution of a bleed as “excellent” or “good” in 84% of all treated bleeding episodes. “Excellent” was defined as a dramatic response with abrupt pain relief and clear reduction in joint or hemorrhage site size. “Good” was defined as pain relief or reduction in hemorrhage site size that may have required an additional infusion for resolution.

Ixinity also induced hemostasis in patients who underwent major surgical procedures.

The drug exhibited similar pharmacokinetic behavior as nonacog alfa, another licensed recombinant coagulation factor IX product. There was no significant reduction in steady-state factor IX levels or alteration in pharmacokinetic behavior over time with Ixinity.

There were 14 adverse events reported in 6 patients. The most common event, observed in 2.6% of patients, was headache. Other adverse events were asthenia, apathy, depression, dysgeusia, influenza, injection site discomfort, lethargy, and skin rash.

None of the patients developed inhibitors to Ixinity, and there were no reports of thrombotic events or allergic reactions.

For more details on this research, see the full prescribing information for Ixinity, available at www.IXINITY.com.

Antihemophilic factor

The US Food and Drug Administration (FDA) has approved an intravenous, recombinant, human coagulation factor IX product (Ixinity) for use in patients

with hemophilia B.

The drug is intended to control and prevent bleeding episodes and for perioperative management in adults and children age 12 and older.

Concurrent with the FDA’s approval, Emergent Biosolutions (the company developing Ixinity) launched the Ixinity IXperience Concierge. This resource, which consumers can access by calling 1-855-IXINITY, provides information on the drug.

About Ixinity

Ixinity contains trenonacog alfa, a purified, single-chain glycoprotein derived from Chinese hamster ovary (CHO) cells that has an amino acid sequence comparable to the Thr148 allelic form of plasma-derived factor IX.

No human or animal proteins are added during any stage of manufacturing or formulation of Ixinity. The recombinant factor IX is purified by a chromatography purification process.

The process includes 3 validated steps for virus inactivation and removal. It also includes a validated manufacturing step to reduce the presence of CHO proteins in the final drug product.

Ixinity is contraindicated in patients who have known hypersensitivity to the drug or its excipients, including CHO protein. Hypersensitivity reactions, including anaphylaxis, may occur following treatment with Ixinity. Patients who receive Ixinity are also at risk of developing nephrotic syndrome and thromboembolism.

Trial data

The FDA approved Ixinity based on results from a phase 1/3 trial of the drug in previously treated adults and children (age 12 and older) with severe to moderately severe (factor IX level < 2%) hemophilia B.

Seventy-seven patients received at least 1 dose of Ixinity. The drug was given as routine prophylaxis or on-demand treatment for bleeding episodes.

Fifty-five patients received treatment for more than 50 exposure days, and 45 received the drug for more than 100 exposure days. The median duration of treatment on study was 16.2 months (range, 2.4-39.6 months) for the routine treatment regimen and 14.1 months (range, 2.3-36.9 months) for the on-demand treatment regimen.

A total of 508 bleeding episodes were treated with Ixinity—286 bleeds for patients on routine treatment and 222 for patients receiving on-demand treatment. A majority of the bleeds (84%) were resolved by 1 or 2 infusions of the drug.

Patients rated hemostatic efficacy at the resolution of a bleed as “excellent” or “good” in 84% of all treated bleeding episodes. “Excellent” was defined as a dramatic response with abrupt pain relief and clear reduction in joint or hemorrhage site size. “Good” was defined as pain relief or reduction in hemorrhage site size that may have required an additional infusion for resolution.

Ixinity also induced hemostasis in patients who underwent major surgical procedures.

The drug exhibited similar pharmacokinetic behavior as nonacog alfa, another licensed recombinant coagulation factor IX product. There was no significant reduction in steady-state factor IX levels or alteration in pharmacokinetic behavior over time with Ixinity.

There were 14 adverse events reported in 6 patients. The most common event, observed in 2.6% of patients, was headache. Other adverse events were asthenia, apathy, depression, dysgeusia, influenza, injection site discomfort, lethargy, and skin rash.

None of the patients developed inhibitors to Ixinity, and there were no reports of thrombotic events or allergic reactions.

For more details on this research, see the full prescribing information for Ixinity, available at www.IXINITY.com.