Note: This is an edited form of a press release from Octapharma. To read the original release in its entirety,Â click 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.