FDA and European Commission Grant Orphan Drug Status to Deupirfenidone for IPF

The FDA and the European Commission granted an orphan drug designation for deupirfenidone to treat idiopathic pulmonary fibrosis (IPF).¹ The therapy is being developed by PureTech Health plc.

Orphan drug designation is intended to support the development of therapies for rare diseases affecting relatively small patient populations. In the US, drugs that receive this designation may qualify for tax credits for eligible clinical trials, exemptions from certain regulatory user fees, and up to 7 years of market exclusivity if the treatment ultimately receives approval.²

These incentives are designed to encourage pharmaceutical companies to invest in therapies targeting diseases with significant unmet medical needs, particularly when limited treatment options exist.^1,2

IPF is a rare, progressive, and ultimately fatal lung disease characterized by scarring and thickening of lung tissue. Over time, this scarring reduces the lungs’ ability to transfer oxygen into the bloodstream, leading to worsening breathing difficulties and declining lung function. Although several therapies have been approved for IPF, treatment options remain limited, and many patients are unable to tolerate current therapies or experience only modest benefits. The new orphan drug designation for deupirfenidone may eventually help expand treatment possibilities for patients living with the disease and improve long-term outcomes.

Deupirfenidone is a next-generation antifibrotic and a deuterated form of pirfenidone, 1 of 3 FDA-approved therapies to treat IPF, with the potential to become a new standard of care to treat IPF. The designation was based on the global phase 2b randomized, double-blind, active- and placebo-controlled, dose-ranging ELEVATE IPF trial (NCT05321420), in which deupirfenidone demonstrated favorable safety and tolerability.

“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,” Robert Lyne, CEO of PureTech Health, said in a press release.

In the trial, 257 patients were randomized 1:1:1:1 to receive deupirfenidone 550 mg, deupirfenidone 825 mg, pirfenidone 825 mg, or a placebo. Those who received 825 mg of pirfenidone 3 times a day experienced a slower rate of lung function decline at 26 weeks compared with patients who received 801 mg of pirfenidone or a placebo (change in absolute forced vital capacity [FVC], –21.5 mL vs –51.6 mL vs –112.5 mL, respectively).¹

After the blinded portion of the trial, approximately 90% of patients enrolled in the open-label extension (OLE). Patients who continued treatment with 825 mg of deupirfenidone experienced an overall FVC decline of –32.8 mL over a 52-week period.

Completion rates in the OLE were also high. At 52 weeks, 88.1% of patients in the deupirfenidone 825-mg arm and 87.2% in the 550-mg arm remained on therapy, indicating favorable tolerability and continued engagement with treatment.³

“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,” Lyne said.

Adverse events were most common in the 550-mg and 825-mg arms. In the 550-mg arm, 7 patients experienced dyspepsia and 6 experienced upper respiratory infection (URI), whereas in the 825-mg arm, 8 patients experienced URI.³

Late-stage development of deupirfenidone is being advanced by Cela Therapeutics, a newly founded entity established by PureTech, with the potential for commercialization.

References

1. PureTech announces orphan drug designations granted by the U.S. Food and Drug Administration and European Commission for deupirfenidone (LYT-100) in idiopathic pulmonary fibrosis. News release. PureTech Health. February 19, 2026. Accessed March 12, 2026. https://news.puretechhealth.com/news-releases/news-release-details/puretech-announces-orphan-drug-designations-granted-us-food-and

2. Designating an orphan drug or biologic. FDA. Accessed March 12, 2026. https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions/designating-orphan-product-drugs-and-biological-products

3. Tzouvelekis A, Hamblin MJ, Choi WI, et al. Late breaking abstract-ELEVATE IPF phase 2b open-label extension demonstrates durable efficacy of deupirfenidone. Eur Respir J. 2025;66(suppl 69):OA4456. doi:10.1183/13993003.congress-2025.OA4456