Gordon Fox • February 9, 2022
Mavacamten instead of septal reduction?
Mavacamten inhibits the movement of myosin, a key component of heart muscle. Jeff16, CC BY-SA 4.0 https://creativecommons.org/licenses/by-sa/4.0 , via Wikimedia Commons

Many people first discover they have HCM because blood flow out of their heart is obstructed. Some of these patients eventually need to have their septums reduced physically by surgery ( septal myectomy ) or alcohol septal ablation. Can a drug replace these invasive treatments?

On April 2, 2022 Dr. Milind Y. Desai , a leading HCM researcher and practitioner, will present initial results on mava camten ‘s use in severely obstructed HCM patients. Dr. Desai is Director of the HCM Center at the Cleveland Clinic. Scheduled for the American College of Cardiology meeting in Washington. His talk on “Mavacamten as an alternative to surgical septal myectomy or alcohol ablation in patients with severely symptomatic obstructive hypertrophic cardiomyopathy” will present results from the VALOR-HCM trial.

This will be the first set of results on mavacamten’s use in HCM patients who are obstructed. For further background on mavacamten, see this interview with Dr. Martin S. Maron.

HCMA Blog

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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Alnylam Receives European Commission Approval for AMVUTTRA® (vutrisiran) for the Treatment of ATTR Amyloidosis with Cardiomyopathy (ATTR-CM)

By Erica Friedman • June 18, 2025
Alnylam press release on the European Commission approval of AMVUTTRA® (vutrisiran) forwild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM) as an additional indication
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Alnylam Announces FDA Approval of AMVUTTRA® (vutrisiran) for Adults with ATTR Amyloidosis with Cardiomyopathy (ATTR-CM)

By Erica Friedman • June 17, 2025
AMVUTTRA® (vutrisiran) has been approved by the U.S. Food and Drug Administration (FDA) to treat ATTR-CM in adults.
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