Alnylam Pharmaceuticals, Inc. announced today that the FDA has granted Orphan Drug Designation to ALN-TTRsc02. This drug has the potential to be a once-quarterly, low volume, subcutaneously administered RNAi medication in the management of ATTR amyloidosis.
The FDA Orphan Drug Designation Program provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S.
The European Commission also recently issued the decision to adopt the opinion of the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) and designate ALN-TTRsc02 as an orphan medicinal product in the European Union (EU) for the treatment of transthyretin-mediated amyloidosis.
Read the press release here.