The FDA has expanded approval of a hemophilia treatment developed by Pfizer to include pediatric patients ages 6-11 with or without inhibitors and for patients 12 and older with inhibitors.
The expanded indication covers routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and pediatric patients age 6 and older with hemophilia A or B with or without inhibitors, according to a June 8 Pfizer news release. The treatment is the first subcutaneous nonfactor therapy available for pediatric patients ages 6-11 with hemophilia B .
Hemophilia affects more than 800,000 people worldwide, according to the release. Inhibitors develop in approximately 20% of people with hemophilia A and 3% of people with hemophilia B, limiting treatment options for some patients.
The approval was supported by interim results from the Phase 3 Basis Kids trial, which evaluated children ages 6-17 with hemophilia A or B with or without inhibitors. Among children ages 6-17 with inhibitors, the treatment was associated with a mean treated annualized bleeding rate of 1.4 compared with a historical model-based rate of 18.9.
The FDA also cited results from the Phase 3 Basis trial in adults and adolescents ages 12 years and older with hemophilia A or B with inhibitors. The study found a 93% reduction in mean treated annualized bleeding rate compared with on-demand intravenous treatment with bypassing agents.
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