Note: This is an edited form of a press release from Octapharma. To read the original release in its entirety,┬áclick here.
Octapharma USA announced on September 15, 2015, that the U.S. Food and Drug Administration (FDA) has approved NUWIQ┬«, Antihemophilic Factor (Recombinant), an intravenous therapy for adults and children living with Hemophilia A. The NUWIQ┬« 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 impacts the lives of up to 16,000 individuals in the U.S. and their caregivers. Although present therapies for Hemophilia A treatment exist in the U.S., significant challenges still remain, including development of inhibitors and the need for multiple infusions on a prophylactic basis.
The European Commission first approved the therapy in August 2014. NUWIQ┬« is currently approved in many countries, including the United Kingdom, Australia, Canada, Germany, Italy, Sweden and Argentina.
The initial global clinical study program for NUWIQ┬« commenced with a pharmacokinetic (PK) evaluation in an open-label, multi-center clinical trial of 22 (20 adults, 2 adolescents) previously treated patients (PTPs). In this study, 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 PK results for mean half-life were longer than earlier generations of recombinant FVIII products currently available in the U.S.
The second set of global clinical studies for NUWIQ┬« also evaluated 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, 3 adolescents between ages 12 and 17, and 58 pediatric patients between ages 2 and 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, overall prophylactic efficacy of NUWIQ┬« 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 to 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 in 30 of 30 (100%) bleeds in adults and for 89 of 108 (82%) bleeds in children. For on-demand treatment with NUWIQ┬« in 20 adults and 2 adolescents, efficacy for the treatment of bleeds was excellent or good in 931 of 986 (94%) bleeds. Overall efficacy in surgical prophylaxis was rated excellent or good in 32 of 33 (97%) procedures using NUWIQ┬«.
In all clinical studies, NUWIQ┬« had a total of 7 reported adverse events. Each of these adverse events occurred one time with 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.