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Armed with strong phase III safety data in Japanese patients, Palo Alto, Calif.-based Bridgebio Pharma Inc. is planning to file for Japan approval of its investigational drug acoramidis for a rare heart disorder.
1. In this randomized controlled trial, patients with transthyretin (TTR) amyloid cardiomyopathy exposed to acoramidis had slower disease progression than those on a placebo. 2. There were significantly fewer adverse events in the acoramidis group, along with a higher quality of life. Evidence Rating Level: 1 (Excellent) Study Rundown: TTR is a transport protein with
BridgeBio will receive $500 million in exchange for royalties from sales of acoramidis, a drug currently under FDA review for treating heart complications caused by the rare disease transthyretin amyloidosis. The deal also refinances lending agreements, providing BridgeBio with access to additional capital.
A study shows that patients with transthryretin amyloid cardiomyopathy benefited significantly when they took the drug Acoramidis.