Today Biogen Idec (NASDAQ: BIIB) announced that the U.S. Food and Drug Administration (FDA) has approved ALPROLIX™ [Coagulation Factor IX (Recombinant), Fc Fusion Protein], the first recombinant, DNA derived hemophilia B therapy with prolonged circulation in the body. ALPROLIX is indicated for the control and prevention of bleeding episodes, perioperative (surgical) management and routine prophylaxis in adults and children with hemophilia B. The therapy is shown to reduce bleeding episodes with prophylactic (protective) infusions starting at least a week apart.
The approval of ALPROLIX is the first significant advance in hemophilia B treatment in more than 17 years. The therapy is clinically proven to reduce bleeding episodes with a favorable safety and tolerability profile. It is developed using a process called Fc fusion and is the first hemophilia therapy to demonstrate prolonged circulation in the body, which has been shown in adults and adolescents with hemophilia to extend the time between prophylactic infusions.
"The FDA approval of ALPROLIX is a significant milestone for the hemophilia B community, and represents an important first step in our commitment to transform the care of people with hemophilia," said George A. Scangos, Ph.D., chief executive officer of Biogen Idec. "ALPROLIX offers people with hemophilia B the ability to prevent or reduce bleeding episodes with prophylactic infusions starting at least a week apart. We believe this new therapy will help more people with hemophilia and their caregivers realize the benefits of this treatment approach."
The National Hemophilia Foundation (NHF) recommends routine prophylaxis as optimal for the treatment of people with severe hemophilia. Studies show people with severe hemophilia who follow this type of regimen experience fewer bleeding episodes and their related risks. Complications of bleeding episodes may range from severe swelling and pain to arthritis, joint damage and physical disability.1,2,3
"Hemophilia has a significant impact on people whom it affects, throughout their lives," said Patrick F. Fogarty, M.D.,4 assistant professor of medicine at the Hospital of the University of Pennsylvania, and director, Penn Comprehensive Hemophilia and Thrombosis Program. "ALPROLIX addresses a critical need by allowing people with hemophilia B to maintain factor levels with prophylactic infusions once weekly or once every 10 days. We hope this will facilitate use of prophylactic therapy."
Hemophilia B is a rare, chronic, inherited disorder in which the ability of a person's blood to clot is impaired, which can lead to recurrent and extended bleeding episodes. Therapies for hemophilia B can be administered either on a schedule to help prevent or reduce bleeding episodes (prophylaxis), or to help control a bleeding episode when it occurs (on-demand). According to NHF guidelines, traditional hemophilia B therapy requires prophylactic infusions two or more times a week.
"The hemophilia community is excited about new therapy options for the prophylactic management of hemophilia B," said Val Bias, chief executive officer of the National Hemophilia Foundation. "This approval is a significant step forward because it provides an important new option and expanded choice for the hemophilia B community."
The approval of ALPROLIX is based on results from the global, Phase 3 B-LONG study, as well as interim pharmacokinetic (measurement of the presence of the therapy in a person's body over time) and safety data from the Phase 3 Kids B-LONG study. B-LONG study results showed that adults and adolescents with severe hemophilia B achieved prevention or reduction of bleeding episodes with prophylactic infusions at least a week apart. The study included two prophylaxis regimens - the weekly prophylaxis arm and the individualized-interval prophylaxis arm, in which the dosing interval started at once every 10 days. The overall median dosing interval with individualized-interval prophylaxis was 12.5 days; during the last six months of the study, the median interval was 13.8 days. More than 90 percent of all bleeding episodes were controlled by a single ALPROLIX infusion.
No participants in the B-LONG study developed inhibitors (neutralizing antibodies that may interfere with the activity of the therapy) to ALPROLIX. There were no reports of vascular clots or serious allergic reactions. Across the routine prophylaxis and on-demand therapy arms, adverse reactions were reported in 8.4 percent of participants. These adverse reactions included headache, oral paresthesia (abnormal sensation in the mouth), dizziness, dysgeusia (taste alteration), breath odor, fatigue, infusion site pain, palpitations, obstructive uropathy (an obstructing clot in the urinary collecting system) and hypotension (low blood pressure). Each event occurred in two or fewer study participants.
An interim analysis from an ongoing, multi-center Phase 3 pediatric study - Kids B-LONG -showed no inhibitors were detected, and the increase in half-life (a measure of the time therapy remains in the body) seen with ALPROLIX was consistent with data reported in adults and adolescents.
ALPROLIX was recently approved by Health Canada for the treatment of hemophilia B and is currently under review by regulatory authorities in several other countries, including Australia and Japan.
Biogen Idec plans to make ALPROLIX commercially available to people with hemophilia B in the United States in early May. To help address the hemophilia B community's needs, Biogen Idec is committed to providing equitable access to therapy, as well as a variety of personalized assistance and resources through MyALPROLIX™ Services.