The Food and Drug Administration (FDA) has granted Orphan Drug designation to AG10 (Eidos Therapeutics) for the treatment of transthyretin amyloidosis (ATTR). 

AG10 is an investigational, orally-administered small molecule designed to stabilize tetrameric transthyretin, (TTR), targeting ATTR at the source to halt its progression. By binding the 4-part TTR molecules, AG10 stabilizes the structure and prevents the formation of amyloid fibrils.

The treatment is currently being examined in a Phase 2 trial in patients with ATTR cardiomyopathy. Additionally, the AG10 is being investigated in patients with ATTR polyneuropathy. The Company expects to start Phase 3 studies for ATTR cardiomyopathy and polyneuropathy in the first half of 2019. 

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“We believe that AG10 holds great promise as a disease-modifying therapy for patients with ATTR, and Orphan status will help us develop it as quickly as possible,” said Jonathan Fox, MD, PhD, president of Eidos. 

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