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

The FDA has designated Orphan Drug status to Acceleron Pharma's (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.

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