28 March 2018 - - The European Medicines Agency has granted Cambridge, Massachusetts-based RNA interference therapeutics developer Alnylam Pharmaceuticals, Inc. (NASDAQ: ALNY) access to its priority medicines (PRIME) scheme for lumasiran (ALN-GO1), an investigational RNAi therapeutic targeting glycolate oxidase for the treatment of Primary Hyperoxaluria Type 1, the company said.
Results from Parts A and B of the ongoing Phase 1/2 study of lumasiran formed the basis of the application for PRIME, the purpose of which is to bring treatments to patients faster by enhancing the EMA's support for the development of medicines for diseases where there is an unmet medical need and where early clinical data show potential to benefit patients.
Lumasiran, an investigational RNAi therapeutic targeting glycolate oxidase in development for the treatment of Primary Hyperoxaluria Type 1, is designed to reduce the hepatic levels of the GO enzyme, thereby depleting the substrate necessary for oxalate production, which directly contributes to the pathophysiology of PH1.
Lumasiran utilises Alnylam's enhanced stabilization chemistry, GalNAc-conjugate technology, which enables subcutaneous dosing with increased potency and durability, and a wide therapeutic index.
Alnylam Pharmaceuticals is a biopharmaceutical company that discovers, develops, and commercialises novel therapeutics based on RNA interference. Its pipeline of investigational RNAi therapeutics is focused on genetic medicines, cardio-metabolic diseases, and hepatic infectious diseases.