Alnylam Pharmaceuticals Inc
) recently announced that its candidate ALN-TTR02 has been granted
orphan drug status by the US Food and Drug Administration (FDA).
ALN-TTR02 received orphan drug status to treat patients suffering
from familial amyloidotic polyneuropathy (FAP). The disorder refers
to one of the predominant clinical manifestations of transthyretin
(TTR)-mediated amyloidosis (ATTR).
We note that the orphan drug designation is granted in the US
only to drugs that treat a disease affecting less than 200,000
people in the country. Moreover, the orphan drug status for
ALN-TTR02 provides Alnylam Pharma with seven years of marketing
exclusivity (from the day ALN-TTR02 is cleared by the FDA) in the
US for the indication.
We note that earlier this month, Alnylam announced the
completion of enrollment in its phase I trial with ALN-TTR02. The
company also initiated a phase II trial to evaluate clinical
activity, safety, and tolerability of multiple once-monthly doses
of ALN-TTR02 in patients suffering from ATTR. Apart from Alnylam,
(PFE) is also developing its candidate to treat TTR-FAP.
Apart from ALN-TTR02, Alnylam is also developing ALN-TTRsc, for
the utilization of GalNAc-conjugate delivery technology and
subcutaneous dose administration, into clinical development. The
company intends to file a New Drug Application (IND) for ALN-TTRsc
in the second half of 2012 with data expected in the first half of
2013. Furthermore, the company is developing ALN-RSV01 for the
treatment of respiratory syncytial virus infection; ALN-VSP for the
treatment of liver cancer and ALN-HTT for the treatment of
We currently have a Neutral recommendation on Alnylam. The stock
carries a Zacks #2 Rank (Buy rating) in the short-run.
ALNYLAM PHARMA (ALNY): Free Stock Analysis
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