New FDA-Approved Treatment Brings Relief to ATTR-CM Patients

Transthyretin amyloid cardiomyopathy (ATTR-CM) is a rare, progressive heart disease caused by a buildup of a misfolded protein (TTR) in the heart. Over time, this stiffens the heart and leads to symptoms of heart failure (shortness of breath, fatigue, swelling, etc.). Until now, treatment options have been limited. However, the FDA has approved Amvuttra® (vutrisiran) for ATTR-CM in adults, giving new hope to patients, especially older Black Americans who are at high risk of this disease.

Amvuttra is a cutting-edge RNA interference (RNAi) therapy that “works with the body’s natural system to stop the production of the TTR protein”. It’s given as a simple injection under the skin once every three months, and with TTR production lowered, fewer amyloid deposits form in the heart. This approach differs from older drugs (tafamidis, acoramidis) that only stabilize the protein; Amvuttra “works differently by slowing down the production” of TTR entirely.

ATTR-CM can be hereditary (caused by a gene mutation) or wild-type (age-related). A common mutation (Val122Ile) is especially prevalent in people of West African ancestry. Experts estimate over 1.5 million Black men in the U.S. carry a TTR gene variant linked to ATTR-CM – nearly four percent of Black Americans. Yet many cases are missed because symptoms resemble ordinary high blood pressure or aging. One analyst even calls ATTR-CM an “unrecognized pandemic” among Black men.

Clinical trials of Amvuttra have shown real benefits. In the HELIOS-B Phase 3 study, patients on vutrisiran had about 28 percent fewer deaths or serious heart events than those on placebo. In practical terms, people taking the new drug were roughly 30 percent more likely to be alive and out of the hospital over the trial period. They also walked farther on a six-minute walk test and reported feeling better overall. Dr. Ronald Witteles (Stanford) said the drug helped patients “live longer, experience fewer hospitalizations, and improve how they function and feel.” For example, one patient wrote, “I am now doing OK… I am so grateful for my quick diagnosis and ongoing treatment.”

Cost can be a concern, but Amvuttra is usually covered by insurance. Alnylam reports that 99 percent of patients pay $0 out-of-pocket. Importantly, under Medicare Part B, this injectable treatment carries no copay. Patient assistance programs are also available. Don’t let cost stop you from asking about this therapy if it might help.

Practical Tips & Resources

Track symptoms: Note fatigue, breathlessness, swelling, or tingling. Discuss these with your doctor.
Family history: Mention any relatives with unexplained heart failure or neuropathy.
Ask directly: In your appointment, say, “Could this be amyloidosis (ATTR-CM)?” and request screening if needed.
Understand testing: Diagnosis often involves a safe heart scan (PYP scan) and blood tests. Genetic testing can check for the TTR mutation.
Know the treatment: Amvuttra is an injection every 3 months (not a daily pill).
Check coverage: Amvuttra is covered by Medicare Part B (no copay). Most private plans cover it; ask about assistance programs.
Find support: Visit amyloidosis.org (Amyloidosis Foundation) or arci.org (Amyloidosis Research Consortium) for patient guides. BlackDoctor.org and patient forums share personal stories.
Patient perspective: One patient wrote, “I am now doing OK… I am so grateful for my quick diagnosis and ongoing treatment.”