The Food and Drug Administration (FDA) has approved Wilate (von Willebrand Factor/Coagulation Factor VIII Complex [Human]; Octapharma) for routine prophylaxis to reduce the frequency of bleeding episodes and on-demand treatment and control of bleeding episodes in patients with hemophilia A.
The approval was based on data from the prospective, open-label, multicenter phase 3 trial that assessed the safety and efficacy of Wilate in 55 previously treated patients (n=50 adults; n=5 pediatric patients) with severe hemophilia A for 6 months. Patients were eligible if they had ≥150 previous exposure days to a FVIII concentrate. All patients were evaluated for the efficacy of Wilate 20-40 IU/kg in routine prophylaxis and treatment of bleeding episodes.
Efficacy results for Wilate in routine prophylaxis showed 30 (54.6%) patients experienced 0 bleeding episodes, 12 (21.8%) patients had 1 bleeding episode, 4 (7.3%) patients had 2 bleeding episodes, 4 (7.3%) patients had 3 bleeding episodes, and 5 (9%) patients had ≥5 bleeding episodes. Additionally, the annualized bleeding rates for spontaneous bleeds and for all types of bleeds in adults were 1.67 ± 3.11 (median 0, range 0-11.76) and 2.39 ± 3.77 (median 0, range 0-15.69), respectively; the annualized bleeding rates in pediatric patients were 0 (median 0, range 0-0) and 0.4 ± 0.89 (median 0, range 0-2), respectively.
The study also provided data on the efficacy of Wilate in the treatment of bleeding episodes which was assessed by the patient (together with investigator) using the predefined criteria using an ordinal scale of excellent, good, moderate or none. From a total of 57 bleeding episodes, treatment efficacy demonstrated to be excellent for 16 bleeding episodes, good for 32 bleeding episodes, and moderate for 9 bleeding episodes. Wilate successfully treated 84.2% of all bleeding episodes.
With regards to safety, the most common adverse reactions were pyrexia, hypersensitivity reactions, urticaria, and dizziness.
“We are excited for providers and patients who have been looking forward to the day when Wilate would be indicated for hemophilia A,” said Octapharma USA President Flemming Nielsen.
Wilate is already indicated for adults and adolescents with von Willebrand disease (VWD), for on-demand treatment and control of bleeding episodes, and perioperative management of bleeding.
It is supplied as 500 IU and 1000 IU strengths in 5mL or 10mL single-use vials, respectively.
For more information visit wilateusa.com.