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Alnylam Pharmaceuticals, Inc. Message Board

  • robertvince robertvince Jun 19, 2012 1:00 PM Flag

    Alnylam gets orphan status

    CAMBRIDGE, Mass. (AP) -- Alnylam Pharmaceuticals Inc. said Tuesday that it received special regulatory incentives for a drug designed to treat a potentially fatal neurodegenerative disease.

    Alnylam said its drug candidate ALN-TTR02 received orphan drug status. That means if Alnylam's drug is approved, competing products won't be allowed on the market for up to seven years. The Food and Drug Administration grants orphan drug status to therapies that could treat diseases that affect fewer than 200,000 people in the U.S.

    ALN-TTR02 is designed to treat transthyretin familial amyloid polyneuropathy, or TTR-FAP, a rare condition that is normally treated with a liver transplant. Alnylam said it will report early-stage clinical data for its drug in the third quarter.

    Pfizer Inc. is trying to win FDA approval for a TTR-FAP drug called Vyndaqel. However, the FDA has not cleared the drug, and on Monday it asked Pfizer to run another clinical trial to prove that Vyndaqel works.

    Alnylam shares rose 2 cents to $11.77 in midday trading, while Pfizer stock rose 9 cents to $22.70 per share.

 
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