Atyr to discuss efzofitimod’s future with FDA after Phase 3 sarcoidosis trial
EFZO-FIT study missed its main goal but suggested some clinical benefits
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Atyr Pharma will meet with the U.S. Food and Drug Administration (FDA) next month to review results from the Phase 3 EFZO-FIT clinical trial and discuss the path forward for efzofitimod as a treatment for pulmonary sarcoidosis.
The meeting follows a company announcement last year that the trial did not meet its main goal of showing the therapy was significantly superior to a placebo at reducing the need for standard corticosteroid treatment.
Even so, the company reported some clinical benefits with efzofitimod, including a greater likelihood of completely discontinuing corticosteroids, improvements in quality of life related to overall and lung health, and reduced fatigue.
FDA meeting to review EFZO-FIT results, next steps
“We are ready and look forward to engaging with the [FDA] in mid-April to review the results of the study and determine the path forward for efzofitimod in pulmonary sarcoidosis,” Sanjay S. Shukla, MD, Atyr‘s president and CEO, said in a company press release.
The company said it expects to provide an update after receiving the official meeting minutes.
In pulmonary sarcoidosis, granulomas — small clusters of immune cells — form in the lungs, causing inflammation and scarring. Over time, this can make lung tissue thicker and stiffer, making it harder to breathe.
The first-line treatment for sarcoidosis is oral corticosteroids, which suppress the immune responses and inflammation that drive granuloma formation and tissue damage. However, high doses or long-term use of these medications can cause serious side effects.
Efzofitimod works by modulating the activity of NRP2, a protein found on certain immune cells involved in granuloma formation and inflammation. Given through the bloodstream, it is designed to help reduce inflammation and the formation of granulomas, potentially easing symptoms of pulmonary sarcoidosis.
Phase 3 EFZO-FIT trial evaluated efzofitimod in sarcoidosis
In the global EFZO-FIT study (NCT05415137), 268 adults with pulmonary sarcoidosis received efzofitimod at one of two doses (3 mg/kg or 5 mg/kg) or a placebo every four weeks. The main goal was to assess changes in the mean daily corticosteroid dose after 48 weeks of treatment.
Secondary goals included changes in the King’s Sarcoidosis Questionnaire-Lung score, which measures lung health–related quality of life; forced vital capacity, a measure of lung function; and the proportion of participants able to discontinue corticosteroids.
Top-line results showed that the mean daily corticosteroid dose was lower in participants treated with the higher dose of efzofitimod than in those given the placebo. However, the difference was not statistically significant, meaning it could have occurred by chance.
However, more participants treated with efzofitimod were able to completely discontinue corticosteroids and reported improvements in lung health–related quality of life compared with those given the placebo. Lung function, measured by forced vital capacity, was maintained during treatment.
Additional analyses highlight potential benefits of higher dose
Further analyses showed that the higher dose of the therapy (5 mg/kg) was also linked to significantly greater gains in quality of life related to overall health and significantly greater reductions in fatigue. In addition, this dose was associated with a greater likelihood of completely discontinuing corticosteroids among participants who also showed improvements in lung health–related quality of life at 48 weeks.
“In 2025 we announced results from our Phase 3 EFZO-FIT study of efzofitimod in pulmonary sarcoidosis,” Shukla said. “This marked an important milestone, not only for the broader sarcoidosis community, but also for aTyr, as it was the company’s largest and first Phase 3 study.”
Based on these results, the company scheduled a Type C meeting with the FDA to review EFZO-FIT’s top-line data and discuss the next steps for efzofitimod’s development.
Participants who complete EFZO-FIT may also be eligible to receive efzofitimod through an individual expanded access program, which allows patients to access experimental treatments outside clinical trials when no other options are available.
