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# aTyr Pharma Gets Orphan Status on Efzofitimod
- URL: https://www.fdaweb.com/atyr-pharma-gets-orphan-status-on-efzofitimod/
- Published: 2022-04-15T12:00:00.000Z
- Updated: 2026-09-14T17:40:55.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5151612

FDA has granted aTyr Pharma an orphan drug designation for efzofitimod and its use in treating systemic sclerosis (SSc, also known as scleroderma). The company describes efzofitimod as 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). “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,” the company says.