Acoramidis

Unassigned

New Medicines

Transthyretin amyloid cardiomyopathy (ATTR-CM)

Information

New molecular entity
Eidos Therapeutics
Eidos Therapeutics

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
Phase III Clinical Trials
Yes
Yes

Category

Small molecule transthyretin (TTR) stabiliser that binds to tetrameric TTR molecules and stabilises the structure of TTR, thereby mimicking the mechanism of the protective T119M mutation (rescue mutation) and preventing the formation of amyloid fibrils
ATTR-CM is an underdiagnosed and potentially fatal disease. It’s characterised by deposits of amyloid protein fibrils in the walls of the left ventricle, the main pumping chamber of the heart. There are two types - wild-type and familial [1]. In a US study in 2012, the prevalence rate was 17 per 100,000 person-years and incidence rate 55 per 100,000 person-years [2].
Transthyretin amyloid cardiomyopathy (ATTR-CM)
Oral

Transthyretin amyloid polyneuropathy (ATTR-PN)

Information

Licence extension / variation
Eidos Therapeutics
Eidos Therapeutics

Development and Regulatory status

None
Phase III Clinical Trials
Phase III Clinical Trials
Yes
Yes

Category

Small molecule transthyretin (TTR) stabiliser that binds to tetrameric TTR molecules and stabilises the structure of TTR, thereby mimicking the mechanism of the protective T119M mutation (rescue mutation) and preventing the formation of amyloid fibrils
ATTR is characterised by deposits of amyloid protein fibrils throughout the body: these can disrupt structure and function of affected tissues - ATTR-PN results from damage to the autonomic nervous system. Estimated European incidence is <1 in 100,000 and there are thought to be about 150 affected individuals in the UK [2].
Transthyretin amyloid polyneuropathy (ATTR-PN)
Oral