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PureTech Announces Orphan Drug Designations Granted by the U.S. Food and Drug Administration and European Commission for Deupirfenidone (LYT-100) in Idiopathic Pulmonary Fibrosis

PRTC
  • PureTech Health plc announced that the U.S. FDA & European Commission granted Orphan Drug Designation to deupirfenidone (LYT-100) for treating idiopathic pulmonary fibrosis (IPF).
  • Deupirfenidone is being advanced by Celea Therapeutics, a Founded Entity established by PureTech for late-stage development & potential commercialization.
  • Orphan Drug Designation offers market exclusivity & development incentives for therapies targeting rare diseases affecting fewer than 200,000 U.S. patients or fewer than 5 in 10,000 EU individuals.
  • Phase 2b ELEVATE IPF trial results showed deupirfenidone 825 mg three times daily slowed lung function decline (-21.5 mL FVC) significantly more than pirfenidone (-51.6 mL) or placebo (-112.5 mL) at 26 weeks.
  • Open-label extension data indicated deupirfenidone maintained a robust treatment effect over 52 weeks, with an overall FVC decline of -32.8 mL, mirroring natural decline in healthy older adults.
  • Celea Therapeutics plans to finalize financing in the first half of 2026 to initiate the Phase 3 SURPASS-IPF trial, which will compare deupirfenidone to pirfenidone in a head-to-head superiority study.
  • PureTech believes successful Phase 3 SURPASS-IPF results could complete the data package required to support potential registration of deupirfenidone.
  • Deupirfenidone is a next-generation antifibrotic & deuterated form of pirfenidone designed to overcome the tolerability & efficacy tradeoffs limiting current IPF treatments.
  • IPF is a rare, progressive, & fatal lung disease with a median survival of two to five years & no current cure.
  • PureTech intends to use its capital-efficient R&D model to advance deupirfenidone through dedicated structures backed by external capital.