FDA Approves Adynovate to Treat Hemophilia A
- By BSTQ Staff
The U.S. Food and Drug Administration (FDA) has approved Baxalta’s Adynovate (antihemophilic factor [recombinant] PEGylated) for use in adults and adolescents aged 12 years and older with hemophilia A. Adynovate is approved for on-demand treatment and control of bleeding episodes, as well as prophylaxis. Consisting of the full-length coagulation factor VIII molecule (historically known as antihemophilic factor) linked to other molecules known as polyethylene glycol (PEGylated), it is modified to have longer circulating half-life and potentially requires fewer injections than unmodified antihemophilic factor when used to reduce the frequency of bleeding.
FDA approval of Adynovate was based in part on a Phase III clinical trial of patients aged 12 years to 65 years who were assigned to either twice-weekly prophylaxis (40-50 IU/kg) or on-demand treatment (10-60 IU/kg) with the drug. The study found that previously treated patients in a twice-weekly prophylaxis arm had 95 percent fewer annual bleeds compared with those treated on demand (median annual bleed rate was 1.9 vs. 41.5, respectively). During the study, 38 percent of prophylaxis-treated patients experienced zero bleeds, and 57 percent of patients experienced zero joint bleeds based on six months of prophylaxis. Nearly all patients (98 percent) on prophylaxis with Adynovate did not have a dose adjustment in the study, and nearly all bleeding episodes (96 percent) were controlled with one or two infusions of the drug. Common adverse reactions reported in the trial were headache and nausea.
