Alnylam Gets Fast Track Status For Patisiran To Treat ATTR Patients With FAP

(RTTNews) - RNAi therapeutics company Alnylam Pharmaceuticals, Inc. (ALNY) Monday said in a statement that the U.S. Food and Drug Administration, or FDA, has granted Fast Track designation to patisiran for the treatment of transthyretin-familial amyloid polyneuropathy (FAP).

The company says that the Fask Track process is designed to facilitate the development and expediate the review of drugs to treat serious conditions and fill an unmet medical need and to get important new drugs to the patient earlier.

Transthyretin-mediated amyloidosis is an inherited, progressively debilitating, and fatal disease caused by mutations in the TTR gene. The company recently announced positive Phase 2 data, showing that multiple doses of patisiran led to "robust and statistically significant" knockdown of serum TTR protein levels of up to 96 percent, with mean levels of TTR knockdown exceeding 85 percent.

In addition, the company stated that patisiran was found to be generally safe and well tolerated in its study.

"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," said Saraswathy Nochur, Senior Vice President of Regulatory Affairs and Quality Assurance at Alnylam.

Friday, the stock closed at $52.98 on the Nasdaq.