BRUSSELS/FRANKFURT/PARIS (dpa-AFX) - Alnylam Pharmaceuticals, Inc. (ALNY) announced Monday that the European Commission or EC has granted approval for AMVUTTRA (vutrisiran) for the treatment of wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM) as an additional indication.
The RNA interference or RNAi therapeutics company said the approval broadens the indication for its orphan RNAi therapeutic AMVUTTRA.
AMVUTTRA 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.
ATTR-CM is caused by the deposition of misfolded TTR fibrils, which drive progressive and irreversible cardiovascular damage and premature death. AMVUTTRA, an RNAi therapeutic, works upstream by delivering sustained knockdown of disease-causing TTR at its source.
In the EU, AMVUTTRA is administered as a subcutaneous injection once every three months, either by a healthcare professional, or self-administered, offering flexibility in treatment delivery.
The company noted that the EC decision is based on positive results from the pivotal HELIOS-B Phase 3 study - a randomized, double-blind, placebo-controlled, multicenter, global trial in a diverse group of patients reflective of the contemporary ATTR-CM population. The patients included those receiving substantial concurrent use of available standard-of-care therapies such as tafamidis and SGLT2 inhibitors.
In the trial, AMVUTTRA met all 10 pre-specified primary and secondary endpoints across both the overall and monotherapy populations.
In the overall population, AMVUTTRA achieved a 28% reduction in the primary composite of all-cause mortality and recurrent cardiovascular events as compared to placebo.
As per estimates, around 100,000 people are affected by ATTR amyloidosis across Europe, most with cardiomyopathy.
Marianna Fontana, HELIOS-B investigator, Professor of Cardiology, University College London, National Amyloidosis Center, Royal Free Hospital, London, said, 'The HELIOS-B findings provide compelling evidence to support the use of vutrisiran as a first-line treatment option for patients with ATTR-CM.'
In May 2025, the European Medicines Agency's Committee for Orphan Medicinal Products or COMP adopted a positive opinion on the maintenance of the EU Orphan Designation for AMVUTTRA in ATTR amyloidosis.
In the EU, AMVUTTRA is indicated for the treatment of hereditary transthyretin amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy (hATTR-PN); and wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM).
AMVUTTRA was approved by the U.S. Food and Drug Administration and the Brazilian Health Regulatory Agency in March 2025 for the treatment of the cardiomyopathy of wild-type or hereditary ATTR amyloidosis in adults.
Alnylam said it continues to pursue additional global submissions to bring vutrisiran to patients worldwide.
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