FDA grants efzofitimod orphan drug designation for systemic sclerosis
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The FDA has granted an orphan drug designation for biotherapeutics firm aTyr’s potential first-in-class immunomodulator efzofitimod in the treatment of systemic sclerosis, according to a company press release.
In their statement, aTyr said efzofitimod (ATYR1923) 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 (ILD).
“The pathology of SSc-ILD is driven by the same immune cells that are central to sarcoidosis pathology, and NRP2 is upregulated on these cells, particularly on macrophages,” read the press release, in part.
The drug has also been shown to reduce lung and skin fibrosis in animal models of SSc and idiopathic pulmonary fibrosis, where it matched or outperformed known anti-fibrotic agents, including nintedanib (Ofev, Boehringer Ingelheim) and pirfenidone (Esbriet, Genentech), according to aTyr.
“We are very pleased to receive orphan drug designation for efzofitimod for SSc, which marks the second such designation for our efzofitimod clinical program,” Sanjay S. Shukla, MD, MS, president and CEO of aTyr, said in the release. “The data we have presented in animal models of SSc along with the positive findings reported from our recent phase 1b/2a study in pulmonary sarcoidosis patients suggest that efzofitimod has the potential to be a new treatment option that resolves inflammation and subsequent fibrosis in those living with SSc-ILD.
“We look forward to exploring the potential expansion of our efzofitimod clinical program into other forms of ILD with high unmet need where this novel therapeutic may be able to improve patient outcomes.”
The FDA grants orphan designation to support the development of drugs for patients with unmet needs for rare diseases that impact fewer than 200,000 people in the United States. Orphan drug status provides certain benefits, including the potential for 7 years of market exclusivity following regulatory approval, exemption from FDA application fees and tax credits for qualified clinical trials.
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