FDA Approves NUWIQ to Treat Hemophilia A
- By BSTQ Staff
Octapharma’s NUWIQ, antihemophilic factor (recombinant), an intravenous therapy for adults and children living with hemophilia A, has been approved by the U.S. Food and Drug Administration (FDA). The approval includes on-demand treatment and control of bleeding episodes, routine prophylaxis to reduce the frequency of bleeding episodes and perioperative management of bleeding. NUWIQ is the first B-domain deleted recombinant factor VIII derived from a human cell line, not chemically modified or fused with another protein.
The approval is based on positive results of clinical studies. An initial global study for NUWIQ of 22 (20 adults and two adolescents) previously treated patients using a one-stage clotting assay demonstrated a mean half-life of 17.1 hours in adults, 11.9 hours in children ages 2 years to 5 years, and 13.1 hours for children ages 6 years to 12 years. A second set of global clinical studies for NUWIQ evaluated the overall efficacy and tolerability in three prospective, open-label studies in previously treated patients with severe hemophilia A. Across these studies, a total of 135 patients with hemophilia A were treated with NUWIQ, including 74 adults, three adolescents between ages 12 years and 17 years, and 35 pediatric patients between ages 2 years and 11 years. Of these, spontaneous bleeds were rated as excellent or good in 92 percent of 32 adults and 97 percent of 59 children. The mean annualized bleeding rates for spontaneous bleeds during prophylaxis were approximately 1.5 in children and 1.2 in adults. In all studies, there were a total of seven reported adverse events, each of which occurred one time with a rate of 0.7 percent across all 135 patients. Events included paresthesia, headache, injection site inflammation, injection site pain, back pain, vertigo and dry mouth.
