PureTech's Deupirfenidone Receives FDA and EU Orphan Drug Designation for IPF
PureTech Health's deupirfenidone has received Orphan Drug Designation from both the U.S. FDA and European Commission for treating idiopathic pulmonary fibrosis, a rare and fatal lung disease. Phase 2b trial results showed superior efficacy compared to existing therapies.
PureTech Health plc (Nasdaq: PRTC, LSE: PRTC) announced that the U.S. Food and Drug Administration and European Commission have granted Orphan Drug Designation to deupirfenidone (LYT-100) for the treatment of idiopathic pulmonary fibrosis (IPF), a rare, progressive, and fatal lung disease. Deupirfenidone is being advanced by Celea Therapeutics, a Founded Entity established by PureTech to lead its late-stage development and potential commercialization.
Orphan Drug Designation is intended to support the development of therapies for rare diseases, defined as conditions affecting fewer than 200,000 people in the United States or fewer than 5 in 10,000 individuals in the European Union. These designations provide sponsors with a range of incentives intended to encourage the development of medicines for diseases with high unmet medical needs, including market exclusivity upon approval.
IPF is characterized by irreversible scarring of lung tissue, with a median survival of two to five years after diagnosis. Only about 25% of IPF patients in the U.S. had received treatment as of 2019, largely due to tolerability issues with existing medications. Deupirfenidone is a deuterated form of pirfenidone, one of three FDA-approved therapies for IPF.
Results from the global Phase 2b randomized, double-blind, active- and placebo-controlled, dose-ranging ELEVATE IPF trial underscored the differentiated profile of deupirfenidone. In that trial, participants treated with deupirfenidone 825 mg three times a day (TID) experienced a slower rate of lung function decline, as measured by change from baseline of Forced Vital Capacity (FVC), at 26 weeks versus those who were treated with the FDA-approved dose of pirfenidone 801 mg TID or placebo (-21.5 mL vs. -51.6 mL vs. -112.5 mL, respectively), with a 91 mL difference between deupirfenidone 825 mg and placebo at 26 weeks (p = 0.02).
Following the completion of the blinded portion of the trial, 90% of trial completers (170 participants) enrolled in the open-label extension. Those who continued treatment with deupirfenidone 825 mg TID maintained a robust treatment effect and experienced an overall FVC decline of -32.8 mL over a 52-week period, which is similar to the expected natural decline in lung function in healthy older adults over that time (approximately -30.0 mL to -50.0 mL).
Celea Therapeutics intends to finalize financing in the first half of 2026 to support the initiation of the Phase 3 SURPASS-IPF trial in the first half of 2026. SURPASS-IPF will compare deupirfenidone 825 mg TID to pirfenidone 801 mg TID in a head-to-head study powered to test for superiority. Based on feedback from the FDA and other global health authorities, PureTech believes that the results from this single Phase 3 trial, if successful, and supported by the totality of data from the overall deupirfenidone development program, could complete the data package required to support potential registration of deupirfenidone.