Alnylam Pharmaceuticals, an leading RNAi therapeutics company, announced updated positive results from its Phase 1/2 clinical study of Lumasiran, an investigational, subcutaneously administered RNAi therapeutic targeting glycolate oxidase (GO) for the treatment of primary hyperoxaluria type 1 (PH1). Results were presented at the 2018 European Society for Paediatric Nephrology (ESPN) Annual Meeting on October 4…
U.K. Agency Grants Early Access To Alnylam’s Patisiran
Alnylam Pharmaceuticals, an RNAi therapeutics company, announced that the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) has granted Patisiran, an investigational RNAi therapeutic in development for the treatment of hATTR amyloidosis, a positive scientific opinion through the Early Access to Medicines Scheme (EAMS). With this decision, eligible adults in the UK with hATTR amyloidosis…
Neurodegenerative Disease Treatment Receives Breakthrough Therapy Designation
U.S. Food and Drug Administration (FDA) grants Alnylam Breakthrough Therapy Designation (BTD) for patisiran for the treatment of hereditary ATTR (hATTR) amyloidosis with polyneuropathy. Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for patisiran, an investigational RNAi therapeutic targeting transthyretin (TTR) for the treatment…