Octapharma Announces U.S. Availability of NUWIQ® To Treat Adults and Children with Hemophilia A05.01.2016
HOBOKEN, N.J. (January 4th, 2016) – Octapharma USA today announced NUWIQ® , Antihemophilic Factor (Recombinant), is now commercially available. NUWIQ® is indicated for the treatment and control of bleeding, perioperative (surgical) management, and routine prophylaxis to reduce the frequency of bleeding episodes, in adults and children with Hemophilia A.
NUWIQ® is the first and only fourth generation recombinant Factor VIII (FVIII) produced in a human cell line without any chemical modification or protein fusion.1 Octapharma is also launching two community support programs designed to facilitate access to this innovative therapy.
The Hemophilia community has seen great advancements in the past 20 years. Despite these advancements, there still remain major medical unmet needs that can lead to significant morbidity. One is the high incidence of inhibitor development, which reduces the efficacy and effectiveness of FVIII therapies leading to more frequent bleeding episodes. As recently presented at the American Society of Hematology Annual Meeting, the SIPPET (Survey of Inhibitors in Plasma-Products Exposed Toddlers) study reported that recombinant FVIII concentrates produced in hamster cell lines were as a class associated with an 87% higher incidence of inhibitor formation in previously untreated patients compared to plasma-derived FVIII concentrates. Another important treatment challenge is the burden of frequent infusions required to prevent bleeding episodes.
To advance Hemophilia A patient care globally, Octapharma is launching NUWIQ®, the first and only fourth generation recombinant FVIII produced in a human cell line without pegylation or protein fusion. Current Hemophilia A therapies that are derived from a hamster cell line produce non-human epitopes, which may lead to increased immunogenicity. NUWIQ® is made from a human cell line that produces only human post-translational modifications, which may reduce the potential for developing inhibitors.2 Octapharma is exploring the value of NUWIQ® to potentially lower immunogenicity in previously untreated patients (PUPs) through its on-going clinical trial GENA-05 (NuProtect).
Octapharma is further investing in NUWIQ® for the benefit of patients with clinical studies of the dosing process (GENA-21 and GENA-21b) to improve protection against bleeding episodes and reduce the frequency of dosing with personalized prophylaxis. In the GENA-21 study, adult Hemophilia A patients were started on infusions every other day or three times per week. Subsequent dosing intervals were then determined based on individual pharmacokinetic (PK) data, which resulted in a median dosing interval of 3.5 days and with 58% of patients on a twice a week or fewer infusion schedule.3 A patient who is moved from dosing every other day to dosing twice a week would have 78 fewer infusions per year.
“NUWIQ® is an innovative recombinant Factor VIII derived from a human cell line, which holds great promise for Hemophilia A patients in two very significant ways, both of which have the potential to reduce treatment-related complications,” said Craig M. Kessler, M.D., Director of the Hemophilia and Thrombosis Treatment Center and Chief of the Division of Coagulation, Georgetown University Hospital and the Lombardi Comprehensive Cancer Center, Washington, D.C. “First, the product’s human origin may benefit patients by potentially decreasing the likelihood of inhibitor development in PUPs compared to current hamster cell based recombinant products. Second, compared to standard every-other-day dosing, the therapy may decrease the number of infusions required by individual patients through improved product half-life.”
NUWIQ® was U.S. Food and Drug Administration (FDA) approved based on a robust clinical program that included many U.S. study sites. These clinical studies indicate NUWIQ® will provide significant benefit to U.S. patients with the product assessed as highly efficacious by controlling bleeding episodes with only one infusion required in nearly 92% of cases, and a median annual bleeding rate (ABR) of 0.90 in adults and 1.9 in children across all trials.3 Although development of inhibitors can occur following the administration of NUWIQ®, no inhibitors or serious adverse events were experienced in the clinical trials.
Octapharma USA, a subsidiary of global human protein products manufacturer Octapharma AG, has developed two bleeding disorders community support programs for NUWIQ® designed to introduce patients and medical providers to the therapy and help defray medical costs for eligible patients.
“Octapharma is genuinely committed to improving the lives of the more than 16,000 patients and caregivers in the U.S. living with Hemophilia A,” said Octapharma USA President Flemming Nielsen. “We realize family out-of-pocket healthcare expenses can frequently be challenging and hope these support programs can make a difference in bringing patients medical care that can improve their quality of life. Coupled with ongoing educational programs, these efforts emphasize Octapharma’s strong belief that patient needs must always come first.”
The NUWIQ® Co-Pay Assistance Program offers eligible patients up to $12,000 per year on the out of pocket costs associated with treatment. To be eligible, patients must be receiving NUWIQ® treatment or have a prescription to begin therapy. Patients must have commercial insurance or be self-pay. The NUWIQ® Free Trial Program allows eligible patients to receive up to six doses not to exceed 20,000 IUs at no cost. For more information on NUWIQ® and the support programs, please visit www.octapharmausa.com or contact the Octapharma Customer Support Center at (317) 644-6423.
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 products 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 – Valentino LA, Negrier C, Kohla G, et al. The first recombinant FVIII produced in human cells – an update on its clinical development programme. Haemophilia 2014; 20: Suppl. 1: 1-9.
2 – Kessler C, Oldenburg, Escuriola C, et al. Spotlight on the human factor: building a foundation for the future of haemophilia A management. Haemophilia 2015; 21: Suppl. 1: 1-12.
3 – 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.
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