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Nuwiq Receives FDA Approval for the Treatment of Hemophilia A
The FDA has approved antihemophilic factor (recombinant) (Nuwiq, Octapharma), an intravenous therapy for adults and children with hemophilia A. 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 (FVIII) derived from a human cell-line, not chemically modified or fused with another protein, designed for the treatment of patients with hemophilia A, congenital FVIII deficiency.
Hemophilia A affects up to 16,000 individuals in the U.S. and their caregivers. Although therapies for hemophilia A exist in the U.S., significant challenges remain, including the development of inhibitors and the need for multiple infusions on a prophylactic basis.
The global clinical study program for Nuwiq started with a pharmacokinetic evaluation in an open-label, multicenter trial involving 20 adult and two 2 adolescent previously treated patients (PTPs). Nuwiq demonstrated a mean half-life of 17.1 hours using a one-stage clotting assay in adults. Nuwiq was also evaluated in children using a one-stage clotting assay, with a mean half-life of 11.9 hours for ages 2 to 5 and a mean half-life of 13.1 hours for ages 6 to 12. These results for mean half-life were longer than earlier generations of recombinant FVIII products available in the U.S.
A second set of global clinical studies evaluated Nuwiq’s overall efficacy and tolerability in three prospective, open-label clinical studies in PTPs with severe hemophilia A. Across all clinical studies, a total of 135 patients with hemophilia A were treated with Nuwiq, including 74 adults, three adolescents (ages 12 to 17), and 58 pediatric patients (ages 2 to 11). These 135 patients were treated with a total of 16,134 infusions over 15,950 exposure days using Nuwiq.
In a study of 32 adults, Nuwiq’s overall prophylactic efficacy for spontaneous bleeds was rated as excellent or good in 92% of patients. In a study of 59 children, prophylactic efficacy for spontaneous bleeds was rated as excellent or good in 97% of patients. The mean annualized bleeding rates (ABR) for spontaneous bleeds during prophylaxis were approximately 1.5 in children and 1.2 in adults. For hemophilia A patients receiving Nuwiq prophylaxis compared with on-demand treatment, the ABR was reduced 96% for adults and 93% for children.
Treatment of breakthrough bleeds during Nuwiq prophylaxis was rated as excellent or good for 30 of 30 bleeds (100%) in adults and for 89 of 108 bleeds (82%) in children. For on-demand treatment with Nuwiq in 20 adults and two adolescents, efficacy for the treatment of bleeds was excellent or good in 931 of 986 bleeds (94%). Overall efficacy in surgical prophylaxis was rated excellent or good in 32 of 33 procedures (97%) using Nuwiq.
In all clinical studies, Nuwiq had a total of seven reported adverse events. Each of these events occurred once, a rate of 0.7% across all 135 patients. These events were parathesia, headache, injection-site inflammation, injection-site pain, back pain, vertigo, and dry mouth.
Source: Octapharma; September 14, 2015.