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Alnylam Wins Orphan Drug Status

Zacks Equity Research

Alnylam Pharmaceuticals, Inc. (ALNY) recently announced that the U.S. Food & Drug Administration (:FDA) granted orphan drug designation to ALN-AT3 for the hemophilia A indication. We remind investors that last week, the U.S. regulatory body had granted similar status to the candidate for the hemophilia B indication.

ALN-AT3 is a subcutaneously administered ribo nucleic acid interference (RNAi) therapeutic, which targets antithrombin (AT). The candidate is being developed for the treatment of hemophilia (hemophilia A, hemophilia B, and hemophilia A or B) and other rare bleeding disorders (:RBD).

Alnylam now plans to file an investigational new drug (IND) application for the candidate by the end of this year and also intends to initiate a phase I study on ALN-AT3 by early next year.

We note that the orphan drug designation is given to those drugs and biologics that are meant for the safe and effective treatment, diagnosis or prevention of rare diseases that affect not more than 200,000 people in the U.S.

We are impressed by Alnylam’s efforts to develop its pipeline. In Jul 2013, the company announced positive top-line results from a phase I study on ALN-TTRsc. ALN-TTRsc is a RNAi therapeutic targeting the transthyretin (:TTR) gene that is being developed for the treatment of TTR-mediated amyloidosis (:ATTR).

Alnylam plans to initiate a phase II study on ALN-TTRsc (familial amyloidotic cardiomyopathy) later this year depending on the successful completion of the phase I study. Positive results from the phase II study will lead to the initiation of a phase III study on the candidate in 2014. Alnylam also reported positive data from a phase II study on ALN-TTR02, another candidate in its pipeline being developed for the treatment of ATTR.

Moreover, the company is expecting several study results based on its RNAi therapeutic candidates in the coming quarters. We expect investor focus to remain on the development of its pipeline.

Alnylam, a biopharmaceutical company, presently carries a Zacks Rank #3 (Hold). Other biopharma stocks, such as Actelion Ltd. (ALIOF), Alexion Pharmaceuticals, Inc. (ALXN) and Anthera Pharmaceuticals, Inc. (ANTH), are comparatively well placed. While Actelion carries a Zacks Rank #1 (Strong Buy), Alexion and Anthera carry a Zacks Rank #2 (Buy).

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