FDA grants fast-track designation for efzofitimod in systemic sclerosis-associated ILD
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The FDA has granted fast-track designation for efzofitimod, a potential first-in-class immunomodulator, in the treatment of systemic sclerosis-associated interstitial lung disease, according to a press release from the drug’s manufacturer.
The designation is designed to help facilitate development and expedite the review of therapies that treat serious or life-threatening diseases in which there is an unmet need. Benefits to the fast-track status include more frequent interactions with the FDA throughout the development program and eligibility for accelerated approval, priority review and rolling review.
“This fast-track designation reflects the potential of efzofitimod to address a significant unmet need for patients with SSc-ILD, which is the leading cause of death in scleroderma patients,” Sanjay S. Shukla, MD, MS, president and CEO of aTyr, said in the release.
“As highlighted in the ILD sessions at the recent European Respiratory Society International Congress, it is clear that there is a need for more effective and safer therapies for fibrotic lung diseases, including sarcoidosis and ILD that results from scleroderma,” Shukla added. “We believe this designation further validates efzofitimod and greatly expands the market potential for this first-in-class therapeutic.”
According to the company, efzofitimod (ATYR1923, aTyr) downregulates innate and adaptive immune responses in uncontrolled inflammatory disease states through selective modulation of neuropilin-2 (NRP2). The company added that it had established clinical proof-of-concept for efzofitimod in a phase 1b/2a study in patients with pulmonary sarcoidosis, a form of interstitial lung disease. In addition, aTyr is currently studying efzofitimod in patients with pulmonary sarcoidosis in a global phase 3 study called EFZO-FIT.
Efzofitimod has been shown to reduce lung and skin fibrosis in animal models of SSc and idiopathic pulmonary fibrosis, where it matched or was superior to nintedanib (Ofev, Boehringer Ingelheim) and pirfenidone (Esbriet, Genentech), according to the company.
“The pathology of SSc-ILD is driven by the same immune cells that are central to pulmonary sarcoidosis pathology, and NRP2 is upregulated on these cells,” read the release. “Efzofitimod has also been shown to reduce key pro-inflammatory markers that are central to this pathology in a clinical study in patients with pulmonary sarcoidosis.”
These findings led the FDA to grant an orphan drug designation for efzofitimod in April, the company said.
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