PureTech's IPF Drug Deupirfenidone Gets Orphan Designation in US and EU
PureTech Health's IPF drug deupirfenidone received orphan drug designation from the FDA and European Commission. The designation provides market exclusivity and financial incentives. A phase 3 trial is expected to begin in the first half of 2026.
PureTech Health PLC's drug deupirfenidone, intended to treat idiopathic pulmonary fibrosis (IPF), has been granted orphan drug designation by both the US Food & Drug Administration and the European Commission. Orphan drug designations are granted for drugs treating rare and life-threatening conditions, as their development would not be financially viable otherwise. Such designations come with financial benefits such as tax credits and fee reductions, and 7 years of market exclusivity in the US, which is extended to 10 in the EU.
IPF is a progressive, fatal lung disease with a median survival time after diagnosis of two years, affecting less than 5 in 10,000 people. Deupirfenidone is being advanced by Celea Therapeutics, a PureTech subsidiary. Results from phase 2b testing show the drug appears to suggest it slows "lung function decline to a level that more closely mirrors healthy ageing, without compromising tolerability." Celea's CEO said: "Orphan drug designation further validates both the seriousness of this disease and the importance of advancing our program, which we believe has the potential to redefine treatment expectations for patients living with IPF."
A phase 3 trial is expected to begin in the first half of 2026. If successful, the company said, it could complete the data package required for registration. PureTech shares were up 1.6% to 126.20 pence each on Friday afternoon in London.