Alnylam Receives Positive Opinion for Orphan Drug Designation in the EU for ALN-TTRsc02

Alnylam Pharmaceuticals announced the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has issued a positive opinion recommending ALN-TTRsc02, an investigational, subcutaneously administered RNAi therapeutic, for designation as an orphan medicinal product for the treatment of transthyretin (TTR)-mediated (ATTR) amyloidosis.

"We are very pleased to have received a positive opinion from the EMA COMP on our application for Orphan Drug Designation for ALN-TTRsc02," said Rena Denoncourt, Program Leader, ALN-TTRsc02 Program at Alnylam. "With potent and durable TTR knockdown, we believe ALN-TTRsc02 holds great promise as an investigational, once-quarterly, low volume, subcutaneously administered RNAi therapeutic for the treatment of a broad spectrum of patients with ATTR amyloidosis. We look forward to advancing ALN-TTRsc02 into Phase 3 later this year."

Orphan Drug Designation by the European Commission provides regulatory and financial incentives for companies to develop and market therapies that treat a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the European Union (EU), and where no satisfactory treatment is available. In addition to a 10-year period of marketing exclusivity in the EU after product approval, Orphan Drug Designation provides incentives for companies seeking protocol assistance from the EMA during the product development phase and direct access to centralized marketing authorization.

ALN-TTRsc02 is an investigational, subcutaneously administered RNAi therapeutic targeting transthyretin (TTR) in development for the treatment of ATTR amyloidosis. It is designed to target and silence specific messenger RNA, potentially blocking the production of TTR protein before it is made. This may help to reduce the deposition and facilitate the clearance of TTR amyloid deposits in peripheral tissues and potentially restore function to these tissues. The safety and efficacy of ALN-TTRsc02 have not been evaluated by the U.S. Food and Drug Administration, European Medicines Agency or any other health authorit

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