Thursday 17 September 2015

FDA Approves Octapharma’s NUWIQ To Treat Hemophilia A

by PPN Staff 

The FDA has approved Octapharma’s NUWIQ, antihemophilic factor (recombinant), an IV therapy for adults and children living 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.

Up to 16,000 individuals in the United States have hemophilia A. Although there are already therapies for hemophilia A, significant challenges remain, including development of inhibitors and the need for multiple infusions on a prophylactic basis, the company said.

The initial global clinical study program for NUWIQ commenced with a pharmacokinetic (PK) evaluation in an open-label, multicenter clinical trial of 22 previously treated patients (PTPs). These patients consisted of 20 adults and two adolescents. 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 years and a mean half-life of 13.1 hours for ages 6 to 12 years. These PK results for mean half-life were longer than earlier generations of recombinant FVIII products currently available in the United States.

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. From all clinical studies, 135 patients were treated with NUWIQ, including 74 adults, three adolescents between ages 12 and 17 years and 58 pediatric patients between ages 2 and 11 years. All these 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 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 in all of 30 (100%) bleeds in adults and 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 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.

Octapharma USA will offer hemophilia A patients educational and support services in connection with the introduction of NUWIQ, which should be available by early 2016.

NUWIQ is not indicated for the treatment of vonWillebrand disease.

“We are pleased that the treatment options for adults and children with hemophilia A continue to advance with ever more innovative therapies being approved for the U.S.,”  said Val Bias, CEO of the National Hemophilia Foundation. “The continued commitment to develop life-enhancing products for the bleeding-disorders community is absolutely vital. Empowering patients and providers with treatment options, as well as education and support programs, is extremely important to people living with hemophilia A.”

—From company press materials

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