Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNA interference (RNAi) therapeutics company, today announced that the European Commission (EC) has granted approval for the treatment of wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM) as an additional indication for its orphan RNAi therapeutic AMVUTTRA® (vutrisiran). The approval broadens the indication for AMVUTTRA, which now becomes the first and only RNAi therapeutic approved by the EC for the treatment of the cardiomyopathy manifestations of ATTR amyloidosis and the polyneuropathy manifestations of hereditary transthyretin-mediated amyloidosis (hATTR) in adults.

“The HELIOS-B findings provide compelling evidence to support the use of vutrisiran as a first-line treatment option for patients with ATTR-CM,” said Marianna Fontana, M.D., Ph.D., HELIOS-B investigator, Professor of Cardiology, University College London, National Amyloidosis Center, Royal Free Hospital, London. “As a physician, it is a privilege to see the true impact on patients in the clinic. The trial enrolled a broad population reflective of real-world clinical practice, and that’s what makes the results so meaningful. This is a milestone for patients, who now have a new treatment option that has the potential to significantly improve outcomes of this disease.”

In May 2025, the European Medicines Agency’s Committee for Orphan Medicinal Products (COMP) adopted a positive opinion on the maintenance of the EU Orphan Designation for AMVUTTRA in ATTR amyloidosis.

For more Information, read the full Alnylam press release.