Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to patisiran (ALN-TTR02) for the treatment of transthyretin (TTR)-familial amyloid polyneuropathy (FAP). According to the FDA, 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.
“We are very pleased that the FDA has granted Fast Track designation for patisiran for the treatment of ATTR patients with FAP. Based on our clinical studies to date, we continue to be very encouraged by the clinical activity and safety profile we have observed with patisiran, including up to 96% knockdown of TTR, the disease-causing protein in ATTR. In aggregate, our clinical data support our belief that patisiran has the potential to be best-in-class for the treatment of ATTR patients with polyneuropathy,” said Saraswathy (Sara) Nochur, Ph.D., Senior Vice President, Regulatory Affairs and Quality Assurance at Alnylam. “Patisiran is the lead program in our ‘Alnylam 5x15’ product development and commercialization strategy. We recently initiated our APOLLO Phase III study with patisiran, fulfilling our commitment to advance this potential novel therapy for patients and their caregivers.”
Alnylam is developing patisiran for the treatment of ATTR patients with FAP. The company 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 up to 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 (“OLE”) 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 the APOLLO 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.