Erica Friedman • June 17, 2025
Alnylam Announces FDA Approval of AMVUTTRA® (vutrisiran) for Adults with ATTR Amyloidosis with Cardiomyopathy (ATTR-CM)

AMVUTTRA® (vutrisiran) has been approved by the U.S. Food and Drug Administration (FDA) to treat ATTR-CM in adults. 

AMVUTTRA is an RNAi therapeutic that works upstream to deliver rapid knockdown of TTR, addressing the disease at its source, with only four convenient subcutaneous doses per year. By rapidly knocking down TTR production, AMVUTTRA substantially decreases deposition of TTR fibrils, which form amyloid and cause irreversible cardiovascular damage and premature death in patients with ATTR-CM.


Alnylam is incredibly grateful to the patients who participated in clinical trials to advance the treatment of ATTR-CM, including those who participated in the HELIOS-B study. We’re also thankful to their families who supported them and the incredible efforts of the advocacy community, investigators and study staff for their important contributions.

 

To ensure patients who need AMVUTTRA have access to it, Alnylam offers multiple patient support services and resources through Alnylam Assist®, designed to help patients and their families navigate the treatment journey. As part of Alnylam Assist®, Alnylam offers low or no cost programs for eligible patients and most patients treated with AMVUTTRA pay $0 out of pocket. If you would like to learn more about how Alnylam can support patients, you can visit AlnylamAssist.com/AMVUTTRA.


For more information, please see the full Anylam press release.

For a plain language summary of the full publication, Please visit:  Treatment with vutrisiran in people withtransthyretin amyloidosis with cardiomyopathy:a plain language summary


HCMA Blog

Lisa's Letter - July Newsletter

By Lisa Salberg • July 3, 2025
Summer, greetings to all our big-hearted friends As July approaches, our focus is already on the fall and preparing for some major events, including our annual meeting coming up in October. July will also find us on the west coast in Seattle conducting a regional patient education meeting as part of our big-hearted warrior tour. We have been following the generic drug quality issue in the United States very closely and encourage you to watch the series starting with our Hill briefing in April and following with the additional webinars with our partners at Medshadow and the People's Pharmacy, and of course the ProPublica series of articles. See them all here . We are happy to have a new team member on board - we welcome Pam as our coordinator of both our All Hearts Collaborative and Hearts and Minds project. Over the next few months, you're going to be learning more about these two amazing initiatives and how we are working to provide better services for big hearts regardless of where you live, so we are meeting all of our big-hearted friends where they are. Please stay tuned for updates from these projects coming soon. We are also creating new volunteer opportunities and engagements that we hope will make it easier for you to participate in spreading the message of the importance of diagnosis, the importance of community readiness related to CPR and AED use and, of course, helping patients get to their ultimate diagnosis and getting them on the proper treatment pathways. This July I would like to recognize all of the special birthdays in my family, including HCMA Center of Excellence coordinator, Stacey Titus-brown and my daughter Rebecca Salberg. It's a milestone birthday for Becca - it’s hard to believe I have a 30-year-old child. Wishing you all a happy and healthy summer. Go build some memories!
An official seal in black & red with white letters that say HCMS Recognized Centers of Excellence

July's Topic is COE Care at the HCMA

By Sabrina Cuddy • July 3, 2025
Getting HCM care at a HCMA Recognized Center of Excellence has a positive impact on patient's outcomes. Find out why in the post by Health Educator Sabrina Cuddy

Pro Publica Releases Results of FDA Investigation

By Erica Friedman • June 26, 2025
Investigative journalists Debbie Cenziper, Megan Rose, Brandon Roberts and Irena Hwang from Pro Publica and NPR have concluded a 14-month long investigation into the quality of generic drugs coming into the United States from overseas. Among the many voices that spoke up for American patients was HCMA Founder and CEO, Lisa Salberg who has felt the effects of low-quality drugs personally. Salberg believes that fixing this problem is something we can do, even in this time, when even health care is highly politicized. Read the key takeaways from ProPublica’s 14-month investigation into the FDA’s oversight of foreign drugmakers in Threat in Your Medicine Cabinet: The FDA’s Gamble on America’s Drugs .
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