FDA approves vutrisiran to treat ATTR-CM
Alnylam Pharmaceuticals has received U.S. Food and Drug Administration (FDA) approval for vutrisiran, a subcutaneous injection the company sells under the brand name Amvuttra, to treat adult patients with transthyretin amyloid cardiomyopathy (ATTR-CM). Vutrisiran has previously been approved by the FDA for the treatment of polyneuropathy of hereditary ATTR amyloidosis (hATTR-PN).
The FDA’s decision was largely based on data first published in The New England Journal of Medicine in August 2024.[1] The HELIOS-B clinical trial, funded by Alnylam, included 655 patients randomized to either undergo treatment with vutrisiran or a placebo. Treatment was associated with lower rates of all-cause mortality, recurrent cardiovascular events and multiple benefits in terms of quality of life.
“I would like to extend my deepest gratitude to the patients who participated in our clinical trials, their families and caregivers, the clinical researchers, regulators, and my colleagues at Alnylam who made this approval possible,” Yvonne Greenstreet, MBChB, chief executive officer of Alnylam, said in a statement. “Today represents a significant milestone in our nearly 20 years of partnership with the ATTR amyloidosis community, but we are not stopping here. We will continue to innovate for patients with ATTR amyloidosis so they can live longer, better, healthier lives.”
Treatment with vutrisiran requires four subcutaneous doses per year. Alnylam has noted that the expected cost of the drug is approximately $477,000 per year, the same cost associated with prescribing vutrisiran for hATTR-PN. However, the company emphasized that vutrisiran is covered by insurance providers for up to 99% of patients when prescribed for hATTR-PN and it expects a similar trend with ATTR-CM.
Tracking the ATTR-CM market
Pfizer’s tafamidis (Vyndaqel, Vyndamax) was the only available treatment for ATTR-CM for years before BridgeBio’s acoramidis (Attruby) gained approval in November 2024. Now that three ATTR-CM treatments have been approved, patients have more options than ever before.
Amyloidosis Support Groups, a nonprofit organization focused on reaching and helping patients with cardiac amyloidosis, shared its excitement about this trend.
“Despite recent advances, there remains a significant need for patients living with ATTR-CM and I’ve witnessed, firsthand, the impact that ATTR amyloidosis can have on families, including diminished quality of life and the loss of loved ones,” Muriel Finkel, president of Amyloidosis Support Groups, said in the Alnylam statement. “The availability of this groundbreaking treatment option is a significant moment for patients living with ATTR amyloidosis. It represents a beacon of hope for our community.”
Michael has more than 18 years of experience as a professional writer and editor. He has written at length about cardiology, radiology, artificial intelligence and other key healthcare topics.