Treatment for Transthyretin Amyloidosis Granted Orphan Drug Designation

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. 

“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. 

For more information visit Eidostx.com.