Biogen Idec's Eloctate Earns FDA Approval For Hemophilia A
By Cyndi Root
Biogen Idec announced in a press release that the Food and Drug Administration (FDA) has approved Eloctate, indicated for hemophilia A. The Antihemophilic Factor (Recombinant), Fc fusion protein prevents and controls bleeding in children and adults.
In April 2014, the FDA approved another Biogen Idec hemophilia product Alprolix, an orphan drug for bleeding prevention and control in children and adults with hemophilia B. Regulatory authorities in Canada, Australia, and Japan are reviewing Eloctate for approval. Biogen Idec intends to make Eloctate available in the U.S. in July 2014.
George A. Scangos, CEO of Biogen Idec, said, “The proven ability of Eloctate to provide protection from bleeding episodes with prolonged circulation marks the first significant hemophilia A treatment advance in more than 20 years.”
Eloctate
Eloctate is for routine prophylaxis in adults and children, perioperative (surgical) management, and control of bleeding episodes. Biogen Idec developed Eloctate to prolong the therapy’s time in the body by fusing B-domain deleted factor VIII to the Fc portion of immunoglobulin G subclass 1, or IgG1. The Fc fusion process uses a natural pathway to keep the agent in the body longer. Patients take Eloctate via infusion every three to five days in a recommended dose of 50 IU/kg, adjustable to a dose range of 25 to 65 IU/kg. Clinical trials showed that the agent was effective as a preventative therapy (prophylaxis) and for sudden bleeding episodes. Patients found it tolerable and the FDA found it safe and effective.
The FDA based its approval in part on the Phase III A-LONG clinical study.
The multi-center study evaluated 164 males older than 12 years of age with hemophilia A. Results showed that participants who took Eloctate on a preventative basis achieved a statistically significant reduction of bleeding episodes compared to patients who took treatment when a bleeding episode occurred. Patients who started on a twice-weekly dose and adjusted it experienced an annualized bleeding rate (ABR), or projected number of bleeding episodes per year, of 1.6 ABR. Patients who took a weekly fixed dose of 65 IU/ kg/week had a 3.6 ABR. Patients who took treatment on demand, when bleeding occurred, had a 33.6 ABR. The FDA said in its press release that there were no safety concerns in the trials.
Pediatric studies from 38 boys aged 2 to 11 in the Phase III Kids A-LONG study showed good tolerability and the increase in half-life (a measure of the time therapy remains in the body) was similar to the adult studies. Biogen Idec stated that higher or more frequent doses may be indicated in pediatric patients.