Alnylam Pharmaceuticals has received Fast Track designation from the FDA for patisiran (ALN-TTR02) for the treatment of transthyretin (TTR)-familial amyloid polyneuropathy (FAP). Fast Track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The purpose is to get important new drugs to the patient earlier.

Alnylam recently announced positive Phase II data, which showed that multiple doses of patisiran led to robust and statistically significant knockdown of serum TTR protein levels of as much as 96%, with mean levels of TTR knockdown exceeding 85%. Knockdown of TTR was found to be rapid, dose dependent, and durable, and similar activity was observed toward both wild-type and mutant protein. In addition, patisiran was found to be generally safe and well tolerated in this study. The company has recently initiated an open label extension study for patients that participated in the Phase II study; the study includes a number of clinical endpoints, and initial data are expected to be reported in 2014. The company also recently initiated a Phase III trial of patisiran in ATTR patients with FAP, with the study now open for enrollment.

In 2012, Alnylam entered into an exclusive alliance with Genzyme, a Sanofi company, to develop and commercialize RNAi therapeutics, including patisiran and ALN-TTRsc, for the treatment of ATTR in Japan and the broader Asian-Pacific region. Alnylam plans to develop and commercialize the ALN-TTR program in North and South America, Europe, and rest of the world.