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FDA approves Afstyla to treat patients with hemophilia A
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The FDA approved antihemophilic factor (recombinant), single chain to treat adults and children with hemophilia A.
Antihemophilic factor (recombinant), single chain (Afstyla, CSL Behring) — the first and only antihemophilic factor VIII single-chain therapy — is indicated for routine prophylaxis to reduce the frequency of bleeding episodes, as well as for on-demand treatment and control of bleeding episodes and perioperative management of bleeding. The product is designed for long-lasting bleeding protection with dosing two to three times a week.
The AFFINITY clinical development program included two pivotal studies and one extension open-label multicenter study to evaluate the safety and efficacy of Afstyla in children, adolescents and adults with hemophilia A.
Patients treated with the product prophylactically experienced a median annualized spontaneous bleeding rate of 0. The median annualized bleeding rate was 1.14 in adults and adolescents and 3.69 in children aged younger than 12 years.
Of the 1,195 bleeds treated in the pivotal study, 94% of bleeds in adults and adolescents and 96% of bleed in pediatric patients were controlled with no more than two infusions.
Afstyla demonstrated a strong safety profile with no inhibitors observed, according to a press release. The most common adverse reactions included dizziness and hypersensitivity.
“FDA’s approval of the first recombinant single-chain therapy that offers long-lasting hemostatic efficacy provides an important new treatment option for patients and health care providers as it has been specifically designed for increased molecular stability and duration of action,” Lisa Boggio, MD, a researcher in the AFFINITY program, said in the release. “Recombinant offers patients an opportunity for excellent efficacy with a strong safety profile and twice-weekly dosing — potentially helping patients to fit treatment into their active lives.”