FDA OKs modified antihemophilic factor for hemophilia A

November 16, 2015

FDA has approved recombinant, PEGlyated Antihemophilic Factor (Adynovate, Baxalta) for use in patients who have Hemophilia A.

FDA has approved recombinant, PEGylated Antihemophilic Factor (Adynovate, Baxalta) for use in patients who have hemophilia A.

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According to the National Hemophilia Foundation, hemophilia A is genetic disorder caused by missing or  defective factor VIII. Although it is passed down from parents to offspring, about one-third of cases are caused by a spontaneous mutation, a change in the gene. With hemophilia A, people cannot clot properly to stop bleeding and may experience repeated episodes of serious bleeding, primarily into joints, which is associated with significant pain and swelling. Approximately 16,000 Americans have hemophilia A, a disease that primarily affects males.

Adynovate consists of the full-length Coagulation Factor VIII molecule, also known as Antihemophilic Factor, liked to polyethylene glycol (PEGylated). Adynovate is built on the previously approved, full-length, recombinant Antihemophilic Factor, Advate. PEGylation allows Adynovate to last longer in the blood and potentially require less-frequent injections than the unmodified molecule. Adynovate is approved for on-demand treatment and control of bleeding episodes as well as for prophylaxis of bleeding episodes in adults and adolescents, aged 12 years and older, who have hemophila A.

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“The approval of Adynovate provides an important therapeutic option for use in the care of patients with Hemophilia A and reduces the frequency of Factor VIII infusions needed to avoid bleeding,” said Karen Midthun, MD, director of FDA’s Center for Biologics Evaluation and Research, in a press release.

The safety and efficacy of Adynovate were demonstrated in a phase 3 clinical trial of 137 adults and adolescents, aged 12 years and older with hemophilia A. In the trial, patients were randomly assigned to receive either the twice-weekly prophylactic treatment or the on-demand therapy of Adynovate. Results showed that Adynovate was effective in reducing the number of bleeding episodes during routine care as well as in treating and controlling acute bleeding episodes.

Common adverse reactions that were reported in approximately 1% of clinical trial participants include headache and nausea. Adynovate is contraindicated in patient who have had prior anaphylactic reaction to Adynovate, the parent molecule (Advate), mouse or hamster protein, or excipients of the drug.

Adynovate is expected to be available in the United States in the coming weeks. 

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