Acceleron ACE-1334 nabs U.S. Orphan drug tag for connective tissue disorder

 

• The FDA has designated Orphan Drug status to Acceleron Pharma's (NASDAQ:XLRN) ACE-1334 for the treatment of systemic sclerosis, an autoimmune rheumatic disease characterized by excessive production and accumulation of collagen, called fibrosis, in the skin and internal organs.
• The Company intends to initiate a Phase 1b/Phase 2 trial in patients with systemic sclerosis-associated interstitial lung disease (SSc-ILD) in 2021, an autoimmune connective tissue disorder characterized by immune dysregulation.
• The Orphan Drug tag may provide grant funding toward clinical trial costs, tax advantages, FDA user-fee benefits, and seven years of market exclusivity in the U.S. following marketing approval.
• ACE-1334 is a TGF-beta superfamily-based ligand trap designed to bind and inhibit TGF-beta 1 and 3 ligands but not TGF-beta 2, which are believed to be key signaling factors in the pathogenesis of fibrotic disease.
• https://seekingalpha.com/news/3640270-accelerons-aceminus-1334-nabs-u-s-orphan-drug-tag-for-connective-tissue-disorder