FDA Approves Wilate for Adults and Adolescents With Hemophilia A

The U.S. Food and Drug Administration (FDA) has approved Wilate as a prophylactic (preventive) and on-demand treatment for bleeding episodes in adults and adolescents with hemophilia A.

Wilate, developed and marketed by Octapharma, is an injectable medication that contains human plasma-derived von Willebrand Factor (VWF) and coagulation factor VIII (FVIII), the clotting protein missing or defective in people with hemophilia A.

In 2009, the FDA approved Wilate as an on-demand treatment to control spontaneous bleeding episodes in children and adults with von Willebrand Disease (VWD) — a disorder caused by lacking or having faulty VWF in the blood — and to manage bleeding during surgical procedures.

The agency has accepted Octapharma’s application to extend Wilate’s indication to include patients with hemophilia A. This means that adolescent and adult patients with hemophilia A in the U.S. will have access to Wilate both as a routine prophylactic treatment to prevent bleeding episodes, and as on-demand treatment to control such events.

“Octapharma has been committed to providing U.S. hemophilia A patients with complete access to the company product portfolio since our inception,” Flemming Nielsen, president of Octapharma USA, said in a news release.

“We are excited for providers and patients who have been looking forward to the day when Wilate would be indicated for hemophilia A,” he added.

The approval was largely based on findings from the WIL-27 Phase 3 clinical trial (NCT02954575). This study was designed to gather additional data on the safety and efficacy of Wilate in 55 males, 12 and older, with severe hemophilia A, who had been previously treated with a FVIII concentrate for at least 150 days (five months). Wilate was tested as a prophylactic treatment given over six months.

Overall, 136 adolescent and adult males (11 to 66 years old) with hemophilia A received Wilate in five clinical trials either as a prophylactic strategy, on-demand therapy, or perioperative treatment (bleeding management during surgery).

The most common side effect was fever, which was reported by two patients. Other side effects included itchy skin (pruritus), headache, and sleeping problems, all reported by one patient each.

Two of the patients participating in WIL-27 had an unexplained worsening of their thrombocytosis, a disorder caused by the excessive production of platelets.