30 January 2018 - - The European Medicines Agency has accepted Cambridge, Massachusetts-based RNA interference therapeutics developer Alnylam Pharmaceuticals, Inc.'s (NASDAQ: ALNY) marketing authorisation application (MAA) and initiated its review for patisiran, an investigational RNAi therapeutic targeting transthyretin for the treatment of hereditary ATTR (hATTR) amyloidosis, the company said.
Patisiran, an investigational intravenously administered RNAi therapeutic targeting transthyretin in development for the treatment of hereditary ATTR amyloidosis, is designed to silence specific messenger RNA, potentially blocking the production of TTR protein. This 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.
Alnylam has licenses to Arbutus Biopharma LNP intellectual property for use in RNA interference (RNAi) therapeutic products using LNP technology. By harnessing the natural biological process of RNAi occurring in our cells, a major new class of medicines, known as RNAi therapeutics, is on the horizon.
Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic platform, potently 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.