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Nuwiq Receives FDA Approval for the Treatment of Hemophilia A

Both children and adults can use Octapharma’s antihemophilic factor

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.

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