Experimental Pulmonary Sarcoidosis Treatment Efzofitimod on Fast Track
aTyrPharma is set to begin a Phase 3 clinical trial on the treatment later this year
Efzofitimod, an experimental treatment for pulmonary sarcoidosis that’s set to begin Phase 3 clinical testing later this year, has been granted fast track designation by the U.S. Food and Drug Administration (FDA).
This designation is designed to speed up the development and review of investigational treatments that aim to fill unmet needs in the care of serious conditions. The designation confers certain benefits to efzofitimod’s developer aTyr Pharma, including more frequent communications with the FDA during the development process.
“The Fast Track designation for efzofitimod underscores the significant need for a new therapy that provides clinically meaningful outcomes for patients living with pulmonary sarcoidosis,” Sanjay S. Shukla, MD, aTyr’s president and CEO, said in a press release.
“The designation of Fast Track for efzofitimod is important news for the many sarcoidosis patients who remain on prednisone for controlling their disease,” Robert Baughman, MD, emeritus professor of medicine at the University of Cincinnati, said.
“The last drugs approved by the FDA for sarcoidosis were prednisone and other glucocorticoids in the 1950s and most sarcoidosis patients with chronic disease remain on prednisone, with or without other agents which have not been approved by the FDA,” Baughman said.
Efzofitimod, previously named ATYR1923, is a first-in-class therapy intended to ease inflammation by modulating the activity of an immune cell protein called neuropilin-2 (NRP2). The FDA granted the experimental therapy orphan drug status earlier this year. In a Phase 1/2 clinical trial (NCT03824392), efzofitimod was generally well tolerated and improved lung function and eased symptoms such as cough and fatigue compared to a placebo.
aTyr is now sponsoring a Phase 3 clinical trial called EFZO-FIT (NCT05415137) which plans to enroll 264 adults with pulmonary sarcoidosis ages 18 to 75. Participants are assigned to one of two doses of efzofitimod (3 or 5 mg/kg) or a placebo, given via monthly infusions for about a year.
The main goal of EFZO-FIT is to assess whether efzofitimod lets patients reduce the use of glucocorticoids, which can cause problematic side effects over the long term.
The FDA’s decision to put efzofitimod on the fast track “reinforces the potential of this novel immunomodulator to be a transformative, disease modifying therapy and address a major unmet need for the sarcoidosis community,” according to Shukla.
“We are currently investigating efzofitimod in a global pivotal Phase 3 study called EFZO-FIT and we look forward to the opportunity to work closely with the FDA to potentially expedite the delivery of a new treatment to patients in need,” Shukla said.