FDA Approves Acoramidis for Cardiomyopathy in Transthyretin-Mediated Amyloidosis

The FDA has approved acoramidis to treat adults with cardiomyopathy caused by transthyretin-mediated amyloidosis (ATTR-CM), including both hereditary (variant) and wild-type forms. The approval is based on clinical trial data showing that acoramidis reduced deaths and hospitalizations related to cardiovascular issues over a 30-month period.

ATTR-CM is a progressive disease characterized by protein buildup in the heart muscle, leading to stiffness, reduced blood flow, and eventual heart failure. Acoramidis works by targeting the underlying mechanism of this rare condition.

The efficacy and safety of acoramidis were demonstrated in a multicenter, international, randomized, double-blind, placebo-controlled study involving 611 adult patients with wild-type or hereditary (variant) ATTR-CM. During the 30-month study, the primary endpoint measured all-cause mortality and the cumulative frequency of cardiovascular-related hospitalizations. Patients receiving acoramidis experienced improvement in outcomes, with an 81% survival rate compared to 74% in the placebo group. Acoramidis also reduced the mean annual rate of cardiovascular-related hospitalizations to 0.3 per year, compared to 0.6 per year among those receiving placebo. These findings underscore the potential of acoramidis to enhance survival and reduce the burden of hospitalizations in this patient population.

The treatment is taken orally, twice daily, and was generally well-tolerated in trials. The most common side effects were mild gastrointestinal issues, such as diarrhea and upper abdominal pain, which typically resolved without discontinuing the medication.

Acoramidis received orphan drug designation, recognizing its importance for a rare and serious disease with limited treatment options.

Reference

FDA approves drug for heart disorder caused by transthyretin-mediated amyloidosis. US Food and Drug Administration. November 25, 2024. Accessed December 25, 2024. https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-drug-heart-disorder-caused-transthyretin-mediated-amyloidosis