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news.BridgeBio Pharma has received the Japanese Ministry of Health, Labour and Welfare approval for Beyonttra, a brand name for acoramidis, for treating adults with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM).
The selective small molecule is orally administered. Credit: Sardar Faizan on Unsplash.
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Acoramidis is a selective small molecule that is orally administered.
ATTR-CM is a progressive, restrictive cardiomyopathy that leads to heart failure.
BridgeBio Cardiorenal chief medical officer Dr Jonathan Fox said: “There is significant need for new treatment options for ATTR-CM, a progressive, fatal disease, worldwide.
“This latest approval marks an important development for the ATTR-CM community, and we are pleased that patients living with this condition in Japan now have a new treatment option that offers early and sustained reductions in cardiovascular events.”
The approval of Beyonttra in Japan is based on positive results from a Phase III study conducted in the country by Alexion, AstraZeneca Rare Disease, and from the global ATTRibute-CM Phase III study.
In the Japanese trial, acoramidis was found to be mostly well tolerated, and no mortality was reported throughout the treatment period for 30 months.
The global ATTRibute-CM Phase III trial demonstrated a 42% reduction in composite all-cause mortality (ACM) and recurrent cardiovascular hospitalisation (CVH) events relative to placebo at month 30, and a 50% reduction in the cumulative frequency of CVH events at the same time point.
Alexion, AstraZeneca Rare Disease, which will handle all commercial activities for Beyonttra in Japan, holds an exclusive license with BridgeBio’s affiliate, Eidos Therapeutics, to develop and commercialise acoramidis in Japan.
BridgeBio is set to receive milestone payment of $30m upon approval in the country, along with royalties in the low double digits on sales of acoramidis, with commercialisation efforts planned for the first half of 2025.
