SAN DIEGO, September 1, 2026
aTyr Pharma, Inc. expects to receive a response from the U.S. Food and Drug Administration (FDA) by mid-September 2026 regarding the protocol submitted for a planned global Phase 3 study of efzofitimod in patients with chronic, symptomatic pulmonary sarcoidosis with restrictive lung disease. The company submitted the protocol in June 2026 and said the anticipated regulatory response follows feedback from the FDA on the proposed study. The planned trial is intended to evaluate the efficacy and safety of efzofitimod in patients with moderate to severe pulmonary sarcoidosis, potentially advancing the investigational therapy toward a pivotal-stage development program in a disease with limited treatment options.
aTyr Prepares Global Phase 3 Sarcoidosis Study
The proposed Phase 3 study is designed as a randomized, double-blind, placebo-controlled global trial lasting 54 weeks. The study is expected to enroll approximately 372 patients with symptomatic pulmonary sarcoidosis and restrictive lung disease who are receiving stable background treatment with up to 5.0 mg per day of oral corticosteroids and/or an immunosuppressant. Participants would be randomized equally to receive 5.0 mg/kg efzofitimod or placebo intravenously every three weeks, for a total of 17 doses. Background treatments are expected to remain stable throughout the study. The primary endpoint is planned to assess the change from baseline in forced vital capacity (FVC) at Week 48, while the key secondary endpoint will evaluate the change from baseline in the King’s Sarcoidosis Questionnaire-Lung score at Week 48,
Efzofitimod Targets Inflammation and Fibrosis
Efzofitimod is an investigational biologic immunomodulator being developed for interstitial lung diseases, including pulmonary sarcoidosis. The therapy is derived from tRNA synthetase biology and is designed to selectively modulate activated myeloid cells through neuropilin-2, with the goal of resolving inflammation without broad immune suppression and potentially limiting progression of fibrosis. Pulmonary sarcoidosis is a major form of interstitial lung disease characterized by inflammatory processes that can affect lung function and, in some patients, lead to progressive pulmonary fibrosis. aTyr is also evaluating efzofitimod in the Phase 2 EFZO-CONNECT™ study in patients with systemic sclerosis-related interstitial lung disease, supporting development of the candidate across multiple immune-mediated fibrotic lung conditions.
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FDA Response Could Set Next Development Step
The expected mid-September FDA response will be an important regulatory milestone for aTyr as the company seeks to finalize the design and advance its planned Phase 3 pulmonary sarcoidosis program. If the protocol receives a favorable regulatory response, the company would be positioned to move forward with preparations for the global pivotal study in patients with moderate to severe disease. The proposed development strategy focuses on improving both objective lung function and patient-reported respiratory outcomes while maintaining stable background therapy. With limited disease-modifying treatment options available for pulmonary sarcoidosis, successful advancement of efzofitimod could represent an important step in aTyr’s broader strategy to develop therapies targeting inflammation and fibrosis through its proprietary tRNA synthetase platform.
Source :aTyr Pharma,,press relese
