aTyr Pharma Secures FDA Type C Feedback, Plans June 2026 IND for Efzofitimod Phase 3 in Pulmonary Sarcoidosis

A recalibrated endpoint strategy and an accelerated IND timeline are the immediate operational outputs of aTyr Pharma's Type C meeting with the FDA, which reviewed the Phase 3 EFZO-FIT™ study results for efzofitimod in pulmonary sarcoidosis. For regulatory teams designing programs in interstitial lung disease, the agency's endpoint guidance here is instructive: forced vital capacity (FVC) was confirmed as a direct measure of patient function, while the King's Sarcoidosis Questionnaire (KSQ)-Lung score was accepted as a key secondary endpoint pending further content validation work.

aTyr plans to submit an investigational new drug (IND) application in June 2026 for a new global, randomized, double-blind, placebo-controlled Phase 3 study. The target population is narrowed relative to EFZO-FIT™: patients with chronic, symptomatic pulmonary sarcoidosis with restrictive lung disease, defined as FVC percent predicted at or below 80%. This subgroup showed clinically meaningful FVC benefit versus placebo in EFZO-FIT™, alongside positive trends across multiple patient-reported outcomes including the KSQ-Lung score, providing the evidentiary basis for the refined enrollment criteria.

The dosing regimen is also changing. Based on the FDA benefit-risk discussion, efzofitimod will be administered at 5.0 mg/kg once every three weeks, up from the once-every-four-weeks schedule used in prior trials. The company states that the consistent safety profile observed to date supports the increased exposure, and that additional risk mitigation strategies and safety surveillance will be incorporated into the protocol design.

For regulatory affairs leads, the meeting outcome illustrates how the FDA is approaching endpoint selection in rare pulmonary diseases: patient-reported outcome instruments require content validation before they can anchor a primary endpoint, and functional measures with established clinical meaning carry more immediate regulatory weight. The KSQ-Lung's secondary status in this design is not a demotion but a sequencing decision, with validation work running in parallel.

QA and clinical operations teams preparing for the IND submission should note the compressed timeline: June 2026 is five weeks from the date of this announcement, meaning protocol finalization, manufacturing documentation, and preclinical package assembly are already in execution.

The IND submission date will serve as the first measurable checkpoint for whether aTyr's revised program design meets the agency's documented expectations from the Type C meeting.

Source: aTyr Pharma, Inc. via GlobeNewswire, May 11, 2026. Management conference call and webcast held same day at 4:30 PM ET / 1:30 PM PT.