Therapy Areas: Cardiovascular
FDA Accepts Alnylam NDA, Grants Priority Review Status for Patisiran for Treatment of Hereditary ATTR Amyloidosis
6 February 2018 - - The US Food and Drug Administration has accepted for filing Cambridge, Massachusetts-based RNA interference (RNAi) therapeutics developer Alnylam Pharmaceuticals, Inc.'s (NASDAQ: ALNY) new drug application for patisiran, an investigational RNAi therapeutic targeting transthyretin for the treatment of hereditary ATTR (hATTR) amyloidosis, the company said.
The FDA also granted the company's request for Priority Review and has set an action date of August 11, 2018, under the Prescription Drug User Fee Act (PDUFA).
At this time, the FDA is not planning to hold an advisory committee meeting to discuss this application.
Patisiran is designed to silence specific messenger RNA, potentially blocking the production of TTR protein, which may help to enable the clearance of TTR amyloid deposits in peripheral tissues and potentially restore function to these tissues.
The safety and efficacy of patisiran have not been evaluated by the US Food and Drug Administration or any other health authority.
RNAi is a natural cellular process of gene silencing. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic platform, silence messenger RNA, the genetic precursors that encode for disease-causing proteins, thus preventing them from being made.
Alnylam Pharmaceuticals is a biopharmaceutical company that discovers, develops, and commercializes novel therapeutics based on RNA interference. Its pipeline of investigational RNAi therapeutics is focused on genetic medicines, cardio-metabolic diseases, and hepatic infectious diseases.


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