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Updated: Aug 5, 2025

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Published on: June 8, 2022
Efzofitimod: a novel anti-inflammatory agent for sarcoidosis
Robert P Baughman1, Vis Niranjan2, Gennyne Walker3
1Editor-in-chief, Sarcoidosis, Vasculitis and Diffuse Lung Diseases. BAUGHMRP@UCMAIL.UC.EDU.
Abstract:
Efzofitimod is a first-in-class biologic based on a naturally occurring splice variant of histidyl-tRNA synthetase (HARS) that downregulates immune responses via selective modulation of neuropilin-2 (NRP2). Preclinical data found high expression of NRP2 in sarcoidosis granulomas. Treatment with efzofitimod reduced the granulomatous inflammation induced by P. acnes in an animal model of sarcoidosis. A dose escalating trial of efzofitimod in sarcoidosis with chronic symptomatic pulmonary disease found that treatment with efzofitimod was associated with improved quality of life with a trend towards reduced glucocorticoid use and stable to improved pulmonary function. These studies have led to a large Phase 3 trial of efzofitimod in symptomatic pulmonary sarcoidosis.
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