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

Key Terms

orphan drug designation regulatory

Orphan drug designation is a special status given to medicines developed to treat rare diseases affecting only a small number of people. This status often provides benefits like faster approval processes and financial incentives, making it more attractive for companies to develop these drugs. For investors, it signals potential for exclusive market rights and reduced competition, which can impact the drug’s profitability.

idiopathic pulmonary fibrosis medical

Idiopathic pulmonary fibrosis is a chronic lung disease in which the air‑carrying tissue becomes progressively thickened and scarred for no identifiable reason, making the lungs stiff and less able to move oxygen—similar to a sponge that hardens and loses its pores. It matters to investors because it is life‑limiting with limited effective treatments, so clinical trial outcomes, regulatory approvals, pricing and reimbursement decisions can strongly affect the commercial value of therapies and the financial prospects of companies developing treatments.

forced vital capacity (fvc) medical

Forced vital capacity (FVC) is the total amount of air a person can forcefully exhale after taking the deepest breath possible, measured during a simple breathing test. Investors care because changes in FVC are a common, straightforward measure used in clinical trials and regulatory evaluations for respiratory drugs and devices, acting like a yardstick that shows whether a treatment is improving lung capacity or slowing disease progression.

randomized, double-blind, active- and placebo-controlled medical

A randomized, double-blind, active- and placebo-controlled study is a clinical trial design where participants are assigned by chance to one of several groups, neither participants nor researchers know who gets which treatment, and outcomes are compared against both a placebo (inactive pill) and an active comparator (an existing treatment). That combination reduces bias and lets investors judge whether a drug truly works and is safer or better than current options, which affects approval odds and commercial prospects.

open-label extension medical

An open-label extension is a continuation of a clinical trial where all participants and researchers know which treatment is being given, often after an initial blinded phase. It allows further study of a drug's long-term safety and effectiveness. For investors, it can indicate ongoing interest and confidence in a product's potential, influencing perceptions of its future value.

phase 2b medical

Phase 2b is a stage in the development of a new medicine or treatment where researchers test its effectiveness and safety in a larger group of people. This step helps determine whether the treatment works well enough to move forward and if it has manageable side effects, which is important for investors because successful results can lead to potential approval and market opportunity.

phase 3 medical

Phase 3 is the late-stage clinical testing step for a new drug or medical treatment, where the product is given to large groups of patients to confirm effectiveness, monitor side effects, and compare it to standard care. Successful Phase 3 results are often the final scientific hurdle before regulators decide on approval and market launch—like passing a final exam before graduation—and can sharply change a company's valuation and future revenue prospects.

head-to-head study medical

A head-to-head study directly compares two treatments, drugs, devices, or products to see which performs better on the same measures and in the same conditions. For investors, these studies matter because they show relative effectiveness or safety—like a side-by-side car comparison—helping predict which option is likely to win market share, secure regulatory approvals, or drive sales and valuation changes.

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Designations provide market exclusivity as well as other development incentives

BOSTON--(BUSINESS WIRE)-- PureTech Health plc (Nasdaq: PRTC, LSE: PRTC) (“PureTech” or the “Company”), a hub-and-spoke biotherapeutics company dedicated to giving life to science and transforming innovation into value, today announced that the U.S. Food and Drug Administration (FDA) and European Commission have granted Orphan Drug Designation to deupirfenidone (LYT-100) for the treatment of idiopathic pulmonary fibrosis (IPF), a rare, progressive, and fatal lung disease. Deupirfenidone is being advanced by Celea Therapeutics, a Founded Entity established by PureTech to lead its late-stage development and potential commercialization.

Orphan Drug Designation is intended to support the development of therapies for rare diseases, defined as conditions affecting fewer than 200,000 people in the United States or fewer than 5 in 10,000 individuals in the European Union. These designations provide sponsors with a range of incentives intended to encourage the development of medicines for diseases with high unmet medical needs.

“Orphan Drug Designation from both the FDA and European Commission underscores the urgent need for more effective therapies for people living with IPF,” said Robert Lyne, Chief Executive Officer of PureTech Health. “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.”

“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,” said Sven Dethlefs, PhD, Chief Executive Officer of Celea Therapeutics. “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.”

Results from the global Phase 2b randomized, 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, as measured by change from baseline of Forced Vital Capacity (FVC), at 26 weeks versus those who were treated with the FDA-approved dose of pirfenidone 801 mg TID or placebo (-21.5 mL vs. -51.6 mL vs. -112.5 mL, respectively), with a 91 mL difference between deupirfenidone 825 mg and placebo at 26 weeks (p = 0.02). Following the completion of the blinded portion of the trial, 90% of trial completers (170 participants) enrolled in the open-label extension. Those who continued treatment with deupirfenidone 825 mg TID maintained a robust treatment effect and experienced an overall FVC decline of -32.8 mL over a 52-week period,¹ which is similar to the expected natural decline in lung function in healthy older adults over that time (approximately -30.0 mL to -50.0 mL).²

PureTech’s Founded Entity, Celea Therapeutics, intends to finalize financing in the first half of 2026 to support the initiation of the Phase 3 SURPASS-IPF trial in the first half of 2026. SURPASS-IPF will compare deupirfenidone 825 mg TID to pirfenidone 801 mg TID in a head-to-head study powered to test for superiority. Based on feedback from the FDA and other global health authorities, PureTech believes that the results from this single Phase 3 trial, if successful, and supported by the totality of data from the overall deupirfenidone development program, could complete the data package required to support potential registration of deupirfenidone.

About Orphan Drug Designation
Orphan Drug Designation is intended to encourage the development of medicines for rare diseases that affect relatively small patient populations and often lack effective treatment options. Regulatory authorities such as the U.S. Food and Drug Administration (FDA) and European Commission provide orphan designation to improve the feasibility of rare disease drug development through enhanced regulatory interactions, financial incentives, and, upon approval, defined periods of market exclusivity.

About Deupirfenidone (LYT-100)
Deupirfenidone (LYT-100) is in development as a potential new standard of care for the treatment of idiopathic pulmonary fibrosis (IPF). It is a next generation antifibrotic and a deuterated form of pirfenidone, one of three FDA-approved therapies for IPF. The uptake of and adherence to approved antifibrotics has historically been limited by a tradeoff between modest efficacy and tolerability, and only ~25% of people with IPF in the U.S. had ever received treatment as of 2019.³

Deupirfenidone may overcome these limitations. In the global Phase 2b ELEVATE IPF trial, deupirfenidone demonstrated the potential to stabilize lung function decline over at least 26 weeks as a monotherapy while maintaining a favorable safety and tolerability profile. Initial data from an ongoing open-label extension study suggest this effect may be sustained through at least 52 weeks. These findings support the potential for deupirfenidone to offer a meaningful advance for people living with this progressive and deadly disease. Beyond IPF, deupirfenidone may also address multiple underserved fibrotic conditions, including progressive fibrosing interstitial lung diseases.

About Idiopathic Pulmonary Fibrosis (IPF)
Idiopathic pulmonary fibrosis (IPF) is a rare, progressive, and fatal lung disease characterized by irreversible scarring of lung tissue that leads to a steady decline in lung function. Median survival following diagnosis is estimated to be two to five years, and currently there is no cure.⁴

About Celea Therapeutics
Celea Therapeutics is dedicated to advancing transformative treatments for people with serious respiratory diseases. Drawn from the Latin word for “sky,” the name reflects the company’s mission to rise above the status quo and deliver therapies that change lives. The company’s lead program, deupirfenidone (LYT-100), is a Phase 3-ready therapeutic candidate with the potential to set a new standard of care for idiopathic pulmonary fibrosis (IPF) and other fibrotic lung diseases.

Celea was founded by and is currently a wholly-owned subsidiary of PureTech Health plc (Nasdaq: PRTC, LSE: PRTC), a biotherapeutics company dedicated to giving life to science. PureTech’s innovative R&D model drives the creation of Founded Entities like Celea, enabling the advancement of highly promising medicines to patients in a capital-efficient manner. For more information, please visit www.celeatx.com.

About PureTech Health
PureTech Health is a hub-and-spoke biotherapeutics company dedicated to giving life to science and transforming innovation into value. We do this through a proven, capital-efficient R&D model focused on opportunities with validated pharmacology and untapped potential to address significant patient needs. This strategy has produced dozens of therapeutic candidates, including three that have received U.S. FDA approval. By identifying, shaping, and de-risking these high-conviction assets, and scaling them through dedicated structures backed by external capital, we accelerate their path to patients while creating sustainable value for shareholders.

For more information, visit www.puretechhealth.com or connect with us on LinkedIn and X (formerly Twitter) @puretechh.

Cautionary Note Regarding Forward-Looking Statements
This press release contains statements that are or may be forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements that relate to continued development of and regulatory interactions related to deupirfenidone, the potential of deupirfenidone in IPF and other indications, our expectations around our therapeutic candidates and approach towards addressing major diseases, our plans to advance our programs and deliver on our milestones, our future plans, prospects, developments, and strategies. The forward-looking statements are based on current expectations and are subject to known and unknown risks, uncertainties and other important factors that could cause actual results, performance and achievements to differ materially from current expectations, including, but not limited to, those risks, uncertainties and other important factors described under the caption "Risk Factors" in our Annual Report on Form 20-F for the year ended December 31, 2024 filed with the SEC and in our other regulatory filings. These forward-looking statements are based on assumptions regarding the present and future business strategies of the Company and the environment in which it will operate in the future. Each forward-looking statement speaks only as at the date of this press release. Except as required by law and regulatory requirements, we disclaim any obligation to update or revise these forward-looking statements, whether as a result of new information, future events or otherwise.

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1 Integrated analysis of double-blind (26 weeks) and initial open-label extension data from Phase 2b ELEVATE IPF trial as of May 9, 2025, using a random coefficient regression model with absolute FVC including baseline as response variable and week, treatment and interaction between week and treatment as fixed effect. The analysis was performed based on the predefined Full Analysis Set.

2 Valenzuela, C., Bonella, F., Moor, C., Weimann, G., Miede, C., Stowasser, S., & Maher, T. (2024, September). Decline in forced vital capacity (FVC) in subjects with idiopathic pulmonary fibrosis (IPF) and progressive pulmonary fibrosis (PPF) compared with healthy references [Poster presentation]. European Respiratory Society International Congress, Vienna, Austria; and Luoto, J., Pihlsgård, M., Wollmer, P., & Elmståhl, S. (2019). Relative and absolute lung function change in a general population aged 60–102 years. European Respiratory Journal, 53(3), 1701812. https://doi.org/10.1183/13993003.01812-2017

3 Dempsey, T. M., Payne, S., Sangaralingham, L., Yao, X., Shah, N. D., & Limper, A. H. (2021). Adoption of the antifibrotic medications pirfenidone and nintedanib for patients with idiopathic pulmonary fibrosis. Annals of the American Thoracic Society, 18(7), 1121–1128.

4 Fisher, M., Nathan, S. D., Hill, C., Marshall, J., Dejonckheere, F., Thuresson, P., & Maher, T. M. (2017). Predicting life expectancy for pirfenidone in idiopathic pulmonary fibrosis. Journal of Managed Care & Specialty Pharmacy, 23(3-b Suppl), S17–S24. https://doi.org/10.18553/jmcp.2017.23.3-b.s17

 

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Source: PureTech Health plc