PureTech Health’s deupirfenidone (LYT-100) has received Orphan Drug Designation to treat idiopathic pulmonary fibrosis (IPF).
The designations were granted by the US FDA and the European Commission (EC) for the treatment of IPF, a rare, progressive and fatal lung disease.
Orphan Drug Designation aims to support the development of therapies for rare diseases, providing sponsors with incentives to develop drugs for diseases with high unmet medical needs.
Rare diseases are defined as conditions affecting fewer than 200,000 people in the US or fewer than five in 10,000 individuals in the European Union.
Robert Lyne, CEO at PureTech Health, said: “Orphan Drug Designation from both the FDA and European Commission underscores the urgent need for more effective therapies for people living with IPF.
“Critically, only a minority of patients with this progressive and fatal disease have ever been treated with currently approved therapies, largely due to the tradeoff between tolerability challenges and modest efficacy.
“We believe deupirfenidone represents a potentially transformative option for this underserved population and is a compelling example of how PureTech’s model can advance differentiated medicines toward meaningful patient impact.”
Results from the global phase 2b randomised, 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 at 26 weeks versus those who were treated with the FDA-approved dose of pirfenidone 801 mg TID or placebo.
Sven Dethlefs, CEO at Celea Therapeutics, commented: “The phase 2b data for deupirfenidone suggest a new benchmark for efficacy in IPF, with slowing of lung function decline to a level that more closely mirrors healthy aging, without compromising tolerability.
“Orphan Drug Designation further validates both the seriousness of this disease and the importance of advancing our programme, which we believe has the potential to redefine treatment expectations for patients living with IPF.”
Deupirfenidone is being advanced by Celea Therapeutics, a Founded Entity established by PureTech to lead its late-stage development and potential commercialisation. A phase 3 trial is planned for the first half of 2026.