aTyr Pharma Announces FDA Orphan Drug Designation for Efzofitimod (ATYR1923) for Treatment of Systemic Sclerosis

aTyr Pharma, Inc. (Nasdaq: LIFE), a clinical stage biotherapeutics company engaged in the discovery and development of innovative medicines based on novel biological pathways, today announced that the U.S. Food and Drug Administration (FDA) has granted the company orphan drug designation for its lead therapeutic candidate, efzofitimod, for the treatment of systemic sclerosis (SSc, also known as scleroderma).

Efzofitimod is a potential first-in-class immunomodulator that downregulates innate and adaptive immune responses in uncontrolled inflammatory disease states via selective modulation of neuropilin-2 (NRP2). Clinical proof-of-concept was recently established for efzofitimod in a Phase 1b/2a study in patients with pulmonary sarcoidosis, a major form of interstitial lung disease (ILD). Many patients with SSc may develop associated ILD, known as SSc-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. Furthermore, efzofitimod has 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 and pirfenidone.

“We are very pleased to receive orphan drug designation for efzofitimod for SSc, which marks the second such designation for our efzofitimod clinical program,” said Sanjay S. Shukla, M.D., M.S., President and CEO of aTyr. “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.”

Click here to read the full article at GlobeNewswire

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with unmet medical needs. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.