Beyonttra™ (acoramidis), First ≥90% TTR Stabilizer, Approved in Japan for ATTR-CM

BridgeBio Pharma, Inc., recognized for its focus on genetic diseases, recently announced that the Japanese Ministry of Health, Labour and Welfare has approved acoramidis, marketed as Beyonttra, for treating transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) in adults. Acoramidis is a small molecule that stabilizes transthyretin (TTR) with an efficacy exceeding 90%. Increased serum TTR levels due to enhanced stability have been linked with reduced risks of mortality, both general and cardiovascular, in recent studies. ATTR-CM is a serious condition manifesting as an infiltrative cardiomyopathy that can lead to heart failure.

The approval in Japan stems from encouraging results from a Phase 3 study conducted locally by Alexion, AstraZeneca Rare Disease, along with findings from the international ATTRibute-CM Phase 3 trial. In the Japanese study, acoramidis was well-tolerated, and there were no reported mortalities during the 30-month treatment period. The global ATTRibute-CM trial highlighted several positive outcomes, with acoramidis showing a significant early separation in the time to the first event, such as all-cause mortality (ACM) or cardiovascular-related hospitalization (CVH), compared to placebo within three months. At month 30, there was a notable 42% reduction in the combined ACM and recurrent CVH events relative to placebo, alongside a 50% decrease in the cumulative frequency of CVH incidents.

Beyonttra's commercialization in Japan will be overseen by Alexion, AstraZeneca Rare Disease. BridgeBio will receive a $30 million milestone payment and royalties from sales of Beyonttra in Japan. Commercialization efforts for the treatment are set to begin in the first half of 2025.

ATTR-CM is a progressive and fatal disease that significantly impacts patients worldwide, emphasizing the need for effective therapeutic options. With the approval of acoramidis, patients in Japan will have access to a new treatment that offers sustained reductions in cardiovascular events, bringing hope to those affected by this condition.

BridgeBio Pharma, founded in 2015, is dedicated to discovering and developing transformative medicines for genetic diseases. Acoramidis, marketed as Beyonttra in Japan, is designed to treat both wild-type and variant ATTR-CM in adults. The treatment has generally been well-tolerated, with mild side effects that resolved without discontinuation.

Overall, BridgeBio Pharma's work with acoramidis represents a significant advancement in the treatment of ATTR-CM, providing patients with a promising option that can help manage this severe condition effectively.