Â Note: The following is an edited press release from Octapharma USA. The original release can be read in its entiretyÂ here.
Octapharma USA announced on Monday, January 4, 2016 that 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. 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.
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.