Biogen Idec (BIIB: 51.66 -0.05 -0.10%) recently announced that it is moving its hemophilia A pipeline candidate into advanced stages of clinical development. Biogen’s decision to move the long-acting recombinant Factor VIII candidate, which is being developed with Swedish Orphan Biovitrum, into a registrational study was based on encouraging results from a phase I/IIa study.Results from the open-label, cross-over, multi-center, dose-escalation study showed that the candidate was well-tolerated and had a prolonged half-life compared to Baxter’s (BAX: 43.71 -0.20 -0.46%) Advate. The phase I/IIa study was conducted to assess the safety of the hemophilia candidate at different doses.
The advanced stage study will evaluate the safety, pharmacokinetics and efficacy of the candidate in preventing and treating bleeding in hemophilia A patients. The candidate’s ability to prolong protection from bleeding and reduce the number of injections needed for treatment would be a major advantage over existing therapies.
Hemophilia A is a rare, inherited bleeding disorder in which the blood doesn’t clot normally. Hemophilia A occurs in approximately one of 5,000 male births annually. Biogen and its partner are seeking orphan drug designation for the candidate in the EU. The European Medicines Agency’s Committee for Orphan Medicinal Products recently issued a positive recommendation for the orphan drug designation – a final decision should be out soon.
Biogen has another candidate in its hemophilia program. The long-acting recombinant Factor IX entered into a phase IIb/III trial earlier this year for the treatment of hemophilia B. Factor IX enjoys orphan drug designation in both the US and the EU.
Besides Biogen, several other companies including Inspiration Biopharmaceuticals, Inc., CSL Behring, and PTC Therapeutics are working on developing treatments for hemophilia.
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