Alnylam Receives Orphan Drug Designation from the United States Food & Drug Administration for ALN-TTRsc02

Alnylam Pharmaceuticals (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation to ALN-TTRsc02, an investigational RNAi therapeutic for the treatment of transthyretin-mediated amyloidosis. ALN-TTRsc02 has the potential to be a once-quarterly, low volume, subcutaneously administered RNAi medication in the management of this serious disease.

As quoted in the press release:

“We are very pleased to have received Orphan Drug Designation from the FDA for ALN-TTRsc02, which we believe has the potential to become a meaningful treatment option for people living with ATTR amyloidosis, a progressive, debilitating, and often fatal disease,” said Rena Denoncourt, Program Leader, ALN-TTRsc02 Program at Alnylam. “We look forward to the continued development of this investigational therapeutic, including advancing the program into Phase 3 later this year.”

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.

Click here to read the full press release.