PureTech Health announced that the U.S. Food and Drug Administration and the European Commission have granted orphan drug designation to its deupirfenidone (LYT‑100) for the treatment of idiopathic pulmonary fibrosis (IPF). The designation applies to a rare disease affecting fewer than 200,000 people in the United States and fewer than 5 in 10,000 in the European Union.
The orphan designation provides PureTech with seven‑year market exclusivity in the United States and ten‑year exclusivity in the European Union, along with tax credits, fee waivers, and other development incentives that can accelerate the drug’s path to approval and reduce regulatory risk.
Deupirfenidone is a deuterated form of pirfenidone designed to improve efficacy and tolerability. IPF is a progressive, fatal lung disease with limited treatment options; the only other approved therapies are pirfenidone and nintedanib. By offering a potentially better safety and efficacy profile, deupirfenidone could address a significant unmet need in this patient population.
Celea Therapeutics, PureTech’s founded entity, is preparing to secure external financing for a Phase 3 SURPASS‑IPF trial in the first half of 2026. The trial will compare deupirfenidone head‑to‑head with pirfenidone, and the orphan designation strengthens Celea’s commercial prospects while supporting PureTech’s hub‑and‑spoke model of advancing differentiated medicines.
CEO Robert Lyne highlighted the significance of the designation, noting that it underscores the urgent need for more effective therapies for IPF patients and demonstrates PureTech’s ability to bring differentiated medicines to market. Analysts have maintained a positive outlook on PureTech’s pipeline, reflecting confidence in the company’s strategy.
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