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