CAMBRIDGE, Mass. — Alnylam Pharmaceuticals announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending approval of vutrisiran for the treatment of transthyretin amyloidosis with cardiomyopathy (ATTR-CM) in adults. The decision, based on positive Phase 3 trial results, positions vutrisiran to become the first RNA interference (RNAi) therapeutic approved in Europe for this life-threatening heart condition.
ATTR-CM, caused by the buildup of misfolded transthyretin (TTR) proteins in heart tissue, leads to progressive cardiovascular damage and premature death. Vutrisiran works by reducing TTR production at its source through RNA interference, offering a novel approach to managing the disease. The treatment is administered as a quarterly subcutaneous injection, either by healthcare professionals or self-administered, providing patients flexibility in how they receive care.
The CHMP recommendation follows vutrisiran’s recent regulatory approvals in the United States and Brazil. A final decision from the European Commission is expected by June 2025.
“This positive opinion represents an important milestone in expanding access to vutrisiran for patients with ATTR amyloidosis with cardiomyopathy in Europe,” said Dr. Pushkal Garg, Chief Medical Officer of Alnylam. “In the HELIOS-B study, vutrisiran not only reduced TTR levels but also improved survival, reduced hospitalizations, and preserved patients’ functional status and quality of life.”
The CHMP endorsement is supported by data from the pivotal HELIOS-B Phase 3 study, which evaluated vutrisiran in patients with both wild-type and hereditary forms of ATTR-CM. The global, placebo-controlled trial met all ten primary and secondary endpoints, demonstrating significant reductions in mortality and cardiovascular events. Improvements were also seen in patients’ physical function, quality of life, and heart failure symptoms.
Nearly half of the study participants were on TTR stabilizers, but vutrisiran showed consistent benefits across all patient groups. The therapy was well tolerated, with adverse event rates comparable to placebo. The most common side effects were injection site reactions and mild increases in certain liver enzymes. Full study results were recently published in The New England Journal of Medicine.
Vutrisiran is already approved in Europe under the brand name AMVUTTRA® for hereditary transthyretin-mediated amyloidosis with polyneuropathy. With the potential European approval for cardiomyopathy, the drug could offer a new treatment option for patients with limited alternatives.
In addition to ongoing regulatory reviews in Europe, vutrisiran remains under evaluation by Japan’s Pharmaceuticals and Medical Devices Agency (PMDA), with Alnylam planning further global submissions throughout 2025.
