PureTech Health Receives FDA and EU Orphan Drug Designation

PureTech Health announced that the U.S. Food and Drug Administration and European Commission have granted Orphan Drug Designation to deupirfenidone for the treatment of idiopathic pulmonary fibrosis, 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. 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 experienced a slower rate of lung function decline, as measured by change from baseline of Forced Vital Capacity, at 26 weeks versus those who were treated with the FDA-approved dose of pirfenidone 801 mg TID or placebo, with a 91 mL difference between deupirfenidone 825 mg and placebo at 26 weeks. Following the completion of the blinded portion of the trial, 90% of trial completers 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,1 which is similar to the expected natural decline in lung function in healthy older adults over that time.