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.
Bayer and Broad Institute extend cardiovascular research alliance to advance precision therapies
CirCode Biomed's IND application for HM2002 receives FDA clearance
CeleCor Therapeutics completes Phase 3 trial of new anti-platelet agent for heart attack
Sarepta receives UK approval to continue dosing in ELEVIDYS ENVISION study
Ionis reports positive Phase 3 results for olezarsen in moderate hypertriglyceridemia
Nordic Bioscience launches new Endotrophin assay
AbbVie and ADARx Pharmaceuticals partner on next-generation siRNA therapies
Otsuka's leukaemia drug ponatinib launched on Fangzhou platform
FDA accepts Novo Nordisk's filing application for oral semaglutide 25 mg
Avacta Therapeutics reports positive Phase 1 data for lead candidate
Innovent's limertinib receives Chinese regulatory approval for first-line treatment of lung cancer
Primo Biotechnology's Taoyuan facility granted PIC/S GMP Certification
Renibus Therapeutics completes RBT-1 Phase 3 clinical trial enrolment