Alnylam to Present New HELIOS-B Phase 3 Data on Vutrisiran at Heart Failure 2025 Congress

Alnylam Pharmaceuticals (Nasdaq: ALNY) announced it will present new data from its HELIOS-B Phase 3 study of vutrisiran at the Heart Failure 2025 Congress, taking place May 17–20 in Belgrade. The data highlight vutrisiran’s effectiveness in reducing cardiovascular mortality and hospitalizations in patients with transthyretin amyloid cardiomyopathy (ATTR-CM). Presentations will also cover real-world insights, the TRITON-CM study design for next-gen therapeutic nucresiran, and outcomes in patients who progressed on tafamidis. Vutrisiran is already approved in the U.S., Brazil, and recommended by EMA’s CHMP for ATTR-CM.

Alnylam Receives Positive CHMP Opinion for Vutrisiran in ATTR Amyloidosis with Cardiomyopathy

Alnylam Pharmaceuticals announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion recommending approval of vutrisiran (brand name: AMVUTTRA®) for the treatment of wild-type or hereditary transthyretin amyloidosis with cardiomyopathy (ATTR-CM) in adult patients.

Key Highlights:
- The recommendation is based on results from the HELIOS-B Phase 3 study, where vutrisiran significantly reduced mortality and cardiovascular events, while preserving functional status and quality of life.
- Vutrisiran is administered subcutaneously once every three months and offers a clinically differentiated treatment option.
- The drug has already received approvals in the United States (March 20, 2025) and Brazil (March 31, 2025).
- A decision from the European Commission is expected in June 2025.
- If approved, vutrisiran would become the first and only RNAi therapeutic authorized for ATTR-CM in Europe.

Clinical Insights:
- HELIOS-B met all 10 pre-specified primary and secondary endpoints in both overall and monotherapy populations.
- Benefits included improved survival, fewer hospitalizations, better 6-minute walk test performance, and improved quality of life.
- The safety profile was favorable and consistent with previous studies, with injection site reactions and mild increases in liver enzymes being the most common side effects.

About ATTR-CM:
ATTR-CM is a rapidly progressive and potentially fatal disease caused by the deposition of misfolded transthyretin (TTR) protein fibrils in the heart. There are two forms: hereditary (hATTR) and wild-type (wtATTR). The disease is underdiagnosed and affects tens of thousands of people worldwide.

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that it will report financial results for the first quarter ending March 31, 2025 on Thursday, May 1, 2025, before the U.S. financial markets open.

Alnylam Presents New HELIOS-B Data on Vutrisiran at ACC 2025 Following FDA Approval for ATTR-CM

Alnylam Pharmaceuticals announced new data from its HELIOS-B Phase 3 study evaluating vutrisiran in patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM) at the American College of Cardiology’s 2025 Annual Scientific Session. These results support the recent FDA approval of AMVUTTRA (vutrisiran) as the first RNAi therapeutic approved to reduce cardiovascular death, hospitalizations, and urgent heart failure visits in adults with ATTR-CM.

The study demonstrated vutrisiran’s favorable impact on echocardiographic systolic and diastolic cardiac function, showing improvements in both left and right ventricular performance. Vutrisiran also helped maintain or improve patients’ functional capacity and quality of life, particularly in those with earlier-stage heart failure. The findings highlight the importance of early intervention with vutrisiran and were recently published in the Journal of the American College of Cardiology (JACC).

Vutrisiran, administered quarterly via subcutaneous injection, delivers rapid knockdown of transthyretin to treat ATTR at its source. It is currently approved in over 15 countries for hereditary ATTR polyneuropathy and, more recently in the U.S., for ATTR-CM.