FDA Approves Twice-Weekly Hemophilia A Treatment


Note: The following is an edited version of a press release originally published by Baxalta. The original release can be read here.

Baxalta Incorporated announced on Monday, November 16, 2015, that the U.S. Food and Drug Administration (FDA) has approved ADYNOVATE [Antihemophilic Factor (Recombinant), PEGylated], an extended circulating half-life recombinant Factor VIII (rFVIII) treatment for hemophilia A. ADYNOVATE is built on the full-length ADVATE [Antihemophilic Factor (Recombinant)] molecule, a … treatment for hemophilia A ….
In the pivotal phase 3 clinical trial, which served as the foundation for the approval, ADYNOVATE demonstrated efficacy in treating hemophilia patients through routine prophylaxis as well as for on-demand bleeding episodes. Patients, 12 to 65 years of age, in the prospective, global, multi-center, open label, non-randomized study were assigned to either twice weekly prophylaxis (40-50 IU/kg, n=120) or on-demand treatment (10-60 IU/kg, n=17) with ADYNOVATE. The study found that previously-treated patients in a twice-weekly prophylaxis arm had 95 percent fewer annual bleeds compared to those treated on-demand [median annual bleed rate (ABR) 1.9 vs. 41.5, respectively]. During the study, 38 percent (n=120) of prophylaxis-treated patients experienced zero bleeds. Moreover, 57 percent of patients experienced zero joint bleeds based on six months of prophylaxis with ADYNOVATE.2
Nearly all (98 percent) of patients on prophylaxis with ADYNOVATE did not have a dose adjustment in the study. Nearly all (96 percent) bleeding episodes (n=591) were controlled with one or two infusions of ADYNOVATE. No patients developed inhibitors to the treatment; the most common adverse reactions (=1 percent of subjects) were headache and nausea.2
ADYNOVATE will be available in the United States in the coming weeks. Baxalta continues to invest in ADYNOVATE to expand the product’s value for more patients worldwide. Currently, studies are ongoing in previously treated patients (PTPs) with severe hemophilia A undergoing surgery and in pediatric PTPs under the age of 12 with severe hemophilia A. Additionally, Baxalta will initiate a study in previously-untreated patients (PUPs) with severe hemophilia A. …
Hemophilia A is a challenging chronic disease; treatment regimens require regular infusions to reduce the risk of bleeding. Working closely with their health professionals, many patients continue to seek treatment options that can be better personalized to fit their needs, providing both effective bleed protection and simplified dosing schedules. Today, the disease affects approximately 16,000 people in the United States and more than 400,000 people worldwide. With an estimated 75 percent of people with hemophilia undiagnosed and untreated or undertreated globally,3 Baxalta continues to innovate in order to address some of the greatest challenges associated with hematologic disorders, including hemophilia.
Through a collaboration with Nektar Therapeutics, ADYNOVATE leverages proprietary pegylation technology designed to prolong the amount of FVIII available for use in the body. The technology was selected because it maintains the integrity of the parent molecule (ADVATE) and reduces the speed at which the body clears ADYNOVATE, resulting in increased circulating half-life. This proprietary technology has been used for more than 15 years in a number of approved medicines that treat chronic or serious conditions. In addition to the patents relating to pegylated FVIII, Baxalta has an exclusive license in the field of hemophilia to certain other patents relating to pegylated FVIII proteins. These patents protect Baxalta’s pipeline of extended circulating half-life FVIII products, including ADYNOVATE.