FDA Approves Octapharma’s NUWIQ® for the Treatment of Adults and Children with Hemophilia A14.09.2015
HOBOKEN, N.J. September 15th, 2015: Octapharma USA today announced 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. Octapharma USA is a subsidiary of global human protein products manufacturer Octapharma AG, which develops and manufactures high-purity recombinant and plasma-derived coagulation factor concentrates for patients with bleeding disorders.
“Octapharma has been committed to the bleeding disorders community for many years and its decade-long drive to find solutions for Hemophilia A challenges has never wavered,” said Octapharma USA President Flemming Nielsen. “Early development strategies were integral in the development of NUWIQ® and these initial goals have been realized with the FDA approval. NUWIQ® has demonstrated safety and efficacy in global clinical trials and has the potential to positively impact patients’ quality of life in the years ahead. Octapharma is dedicated to providing life enhancing and saving therapies for Hemophilia A and looks forward to bringing NUWIQ® to the U.S. marketplace.”
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 and Sweden.
“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, chief executive officer of the National Hemophilia Foundation (NHF). “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.”
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®.1
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.
As part of its continuing commitment to the bleeding disorders community, Octapharma USA will offer Hemophilia A patients educational and support services in connection with the introduction of NUWIQ®. Octapharma USA aims to have NUWIQ® available in the U.S. marketplace by early 2016.
NUWIQ®, Antihemophilic Factor (Recombinant) Lyophilized Powder for Solution for Intravenous Injection is a recombinant antihemophilic factor [blood coagulation factor VIII (Factor VIII)] indicated in adults and children with Hemophilia A for on-demand treatment and control of bleeding episodes; perioperative management of bleeding; and routine prophylaxis to reduce the frequency of bleeding episodes. NUWIQ® is not indicated for the treatment of von Willebrand Disease.
Important Safety Information
NUWIQ® is contraindicated in patients who have manifested life-threatening immediate hypersensitivity reactions, including anaphylaxis, to the product or its components. Hypersensitivity reactions, including anaphylaxis, are possible. Should symptoms occur, discontinue NUWIQ® and administer appropriate treatment. Development of Factor VIII neutralizing antibodies (inhibitors) may occur. If expected plasma Factor VIII activity levels are not attained, or if bleeding is not controlled with an appropriate dose, perform an assay that measures Factor VIII inhibitor concentration. Monitor all patients for Factor VIII activity and development of Factor VIII inhibitor antibodies.
The most frequently occurring adverse reactions (0.7%) in clinical trials were paresthesia, headache, injection site inflammation, injection site pain, non-neutralizing anti-Factor VIII antibody formation, back pain, vertigo, and dry mouth. For full prescribing information on NUWIQ® please visit www.octapharmausa.com.
About the Octapharma Group
Headquartered in Lachen, Switzerland, Octapharma is one of the largest human protein product manufacturers in the world and has been committed to patient care and medical innovation since 1983. Its core business is the development and production of human proteins from human plasma and human cell-lines. Octapharma employs approximately 6,000 people worldwide to support the treatment of patients in over 100 countries with products across the following therapeutic areas: Hematology (coagulation disorders), Immunotherapy (immune disorders) and Critical Care. The company’s American subsidiary, Octapharma USA, is located in Hoboken, N.J. Octapharma operates two state-of-the-art production sites licensed by the U.S. Food and Drug Administration (FDA), providing a high level of production flexibility. For more information, please visit www.octapharmausa.com.
1 – Octapharma, Data on file. 2015.
This news release contains forward-looking statements, which include known and unknown risks, uncertainties, and other factors not under the company’s control. The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments. These factors include results of current or pending research and development activities and action by the FDA or other regulatory authorities.