US, EU regulators name deupirfenidone an orphan drug for treating IPF
Developer: Trial testing oral therapy expected to start in coming months
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Regulatory authorities in both the U.S. and the European Union have granted orphan drug status to deupirfenidone (LYT-100), an oral therapy Puretech Health is developing to treat idiopathic pulmonary fibrosis (IPF).
This designation aims to provide extra economic incentives to companies that invest in developing medications for rare diseases. The details of the designation are a bit different in the U.S. and the EU, but in both, this status means that Puretech will be entitled to several years of market exclusivity, free from generic competitors, if deupirfenidone is ultimately approved for IPF.
“Orphan Drug Designation from both the FDA and European Commission underscores the urgent need for more effective therapies for people living with IPF,” Robert Lyne, CEO of Puretech, said in a company press release, which called deupirfenidone a “potentially transformative” medication.
Puretech said it’s now planning a clinical trial — expected to launch in coming months — that will test deupirfenidone against an approved IPF therapy.
IPF, a respiratory disease of unknown cause, is marked by scarring, or fibrosis, in the lungs. Deupirfenidone is a modified version of the active agent in Esbriet (pirfenidone), a medication approved for treating IPF.
Although Esbriet has proven effective for reducing lung fibrosis, it also often causes side effects, such as digestive upset, which have limited the extent to which it can benefit patients.
Deupirfenidone aims to offer a better safety profile, according to its developer, allowing higher doses that can facilitate better anti-fibrotic activity.
“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,” Lyne said. “We believe deupirfenidone represents a potentially transformative option for this underserved population.”
Upcoming trial to test deupirfenidone vs. Esbriet
Puretech last year announced positive data from a Phase 2b clinical trial called ELEVATE IPF (NCT05321420). That study tested deupirfenidone against Esbriet or a placebo in more than 200 people with IPF.
The results showed patients given deupirfenidone had slower decline in lung function after six months, with continued stabilization in these patients seen out to a year. Puretech also reported that rates of digestive side effects were lower with deupirfenidone than Esbriet.
The [trial] 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.
Now Celea Therapeutics, a Puretech spinoff, is making plans for a Phase 3 clinical trial to further test deupirfenidone. The upcoming study, dubbed SURPASS-IPF, is expected to start in the first half of 2026, per the developer. It will directly test deupirfenidone against Esbriet with the goal of proving that the investigational therapy better than the approved medicine at maintaining lung function.
Puretech believes that positive results from the Phase 3 study could support applications seeking approval of deupirfenidone, according to the release.
“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, CEO of Celea. “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.”
