Alnylam Pharmaceuticals Inc. (ALNY) 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, Pfizer (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 Huntington’s disease.

Our take

We currently have a Neutral recommendation on Alnylam. The stock carries a Zacks #2 Rank (Buy rating) in the short-run.

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