Deupirfenidone Receives Orphan Drug Designation from FDA and European Commission for IPF
PureTech Health announced that deupirfenidone has received Orphan Drug Designation from both the U.S. FDA and European Commission for idiopathic pulmonary fibrosis, with a Phase 3 trial planned for the first half of 2026.
PureTech Health plc (NASDAQ:PRTC, LSE:PRTC) announced Thursday that its experimental treatment deupirfenidone has received Orphan Drug Designation from both the U.S. Food and Drug Administration and European Commission for idiopathic pulmonary fibrosis (IPF). The company has a market capitalization of $4.27 billion.
IPF is a rare, progressive and fatal lung disease with limited treatment options. The designations provide market exclusivity and development incentives for rare diseases affecting fewer than 200,000 people in the U.S. or fewer than 5 in 10,000 individuals in the EU.
Deupirfenidone is being developed by Celea Therapeutics, a subsidiary established by PureTech to advance the drug candidate. The company plans to initiate a Phase 3 trial called SURPASS-IPF in the first half of 2026, comparing deupirfenidone to pirfenidone in a head-to-head study.
In a Phase 2b trial, patients receiving deupirfenidone 825 mg three times daily showed slower lung function decline compared to those on pirfenidone or placebo. The company reported that participants experienced an overall decline in forced vital capacity of -32.8 mL over 52 weeks, similar to the natural decline seen in healthy older adults.
"Orphan Drug Designation from both the FDA and European Commission underscores the urgent need for more effective therapies for people living with IPF," said the Chief Executive Officer of PureTech Health, in a press release statement.
According to the company, only about 25% of people with IPF in the U.S. had received treatment as of 2019, largely due to tolerability challenges with existing therapies.
PureTech believes that if successful, results from the planned Phase 3 trial, supported by existing data, could complete the package required for potential registration of deupirfenidone.
PureTech Health announced that the U.S. Food and Drug Administration has endorsed the advancement of its drug candidate, deupirfenidone, into a pivotal Phase 3 trial for idiopathic pulmonary fibrosis. This development follows a successful End-of-Phase 2 meeting, with the FDA also supporting a 505(b)(2) regulatory pathway for the drug, which is being developed by Celea Therapeutics, a PureTech Founded Entity.