Acoramidis FDA Approval Status
Last updated by Judith Stewart, BPharm on Jan 10, 2024.
FDA Approved: No
Generic name: acoramidis
Company: BridgeBio Pharma, Inc.
Treatment for: Transthyretin Amyloid Cardiomyopathy
Acoramidis is an orally-administered, highly potent, small molecule stabilizer of transthyretin (TTR) in development for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM).
- Transthyretin amyloid cardiomyopathy is a potentially fatal disease of the heart muscle caused by a mutation in the transthyretin (TTR) gene, which results in the accumulation of amyloid deposits. When the amyloid deposits build up in the heart, the walls can become stiff, ultimately leading to heart failure.
- Acoramidis is designed to mimic a naturally-occurring variant of the TTR gene (T119M) that is considered a "rescue mutation" due to its ability to prevent or minimize ATTR in individuals carrying mutations in the TTR gene.
- NDA submission is based on positive results from ATTRibute-CM Phase 3 study in which acoramidis treatment demonstrated an 81% absolute survival rate and a 0.29 observed mean annual cardiovascular-related hospitalization (CVH) frequency, as well as improvements for a large proportion of patients on laboratory and functional measures.
Development timeline for acoramidis
Further information
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