The Food and Drug Administration (FDA) has granted Orphan Drug designation to ACE-1334 (Acceleron Pharma, Inc) for the treatment of patients with systemic sclerosis.

ACE-1334 is a transforming growth factor (TGF)-beta superfamily-based ligand trap designed to bind and inhibit TGF-beta 1 and 3 ligands, which are believed to be key signaling factors in the pathogenesis of fibrotic disease.

In preclinical studies, ACE-1334 has been observed to have antifibrotic activity in multiple models of fibrosis. The investigational drug was recently evaluated in an ascending-dose phase 1 trial in healthy volunteers. The Company intends to initiate a phase 1b/2 trial in patients with systemic sclerosis-associated interstitial lung disease (SSc-ILD) in 2021.

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The FDA previously granted Fast Track designation to ACE-1334 for the treatment of SSc-ILD.

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Acceleron receives FDA Orphan Drug designation for ACE-1334 for the treatment of systemic sclerosis. [press release]. Cambridge, MA: Acceleron Pharma; December 1, 2020. 

This article originally appeared on MPR