Biogen Idec on Wednesday said its experimental treatment for patients with Hemophilia B, a rare inherited blood disorder that impairs blood coagulation, met the main goal of controlling bleeding in a late-stage trial.
A single injection of the long-lasting clotting factor, being developed in partnership with Swedish Orphan Biovitrum , controlled bleeding in 90.4 percent of all bleeding episodes.
Biogen said it plans to submit an approval application to the US Food and Drug Administration in the first half of 2013.
The study, dubbed B-LONG, enrolled 123 male patients age 12 and older, divided into four groups that were treated at different intervals. Overall, 93.5 percent of patients completed the study.
The companies said the treatment was generally well tolerated, but one serious adverse event was reported. The most common adverse events were nasopharyngitis, influenza, joint pain, upper respiratory infection, hypertension and headache.
They said they expect to file an application with European health authorities after completion of a study in children younger than 12 years old, as required by the European Medicines Agency.
About Hemophilia B
Hemophilia B is a rare, inherited disorder in which the ability of a person's blood to clot is impaired.
Hemophilia B occurs in about one in 25,000 male births annually and is caused by having substantially reduced or no Factor IX activity, which is needed for normal blood clotting.
People with hemophilia B therefore need injections of Factor IX to restore the coagulation process and prevent frequent bleeds that could otherwise lead to pain, irreversible joint damage and life-threatening hemorrhages.
The Medical and Scientific Advisory Council of the National Hemophilia Foundation recommends prophylaxis as the optimal therapy for people with severe hemophilia B.
Currently, prophylaxis in hemophilia B typically requires injections up to three times per week to maintain a sufficient circulating level of clotting factor.