The Food and Drug Administration (FDA) has granted Orphan Drug designation to ATYR1923 for the treatment of sarcoidosis.

Sarcoidosis is a rare inflammatory disease characterized by the formation of granulomas in the body, most commonly in the lungs and lymph nodes. ATYR1923 is a potential first-in-class selective modulator of neuropilin-2 (NRP2) that works by downregulating innate and adaptive immune responses in uncontrolled inflammatory disease states. The Company believes that ATYR1923 can treat sarcoidosis by selectively binding to NRP2 on target immune cells during inflammation, including sarcoid granulomas of the lung and skin. 

Results from a phase 1/2 study (ClinicalTrials Identifier: NCT03824392) in patients with pulmonary sarcoidosis showed that ATYR1923 was safe and well tolerated, with no drug-related adverse events. Additionally, the study demonstrated consistent dose response with trends of benefit on key efficacy endpoints including measures of steroid reduction, lung function, and sarcoidosis symptom measures and inflammatory biomarkers. 

“By reducing steroid burden while improving lung function and measures of sarcoidosis symptoms, ATYR1923 has the potential to be a transformative, disease modifying therapy with clinically meaningful outcomes for patients,” said Sanjay S. Shukla, MD, MS, President and CEO of aTyr. “We look forward to initiating a registrational trial in pulmonary sarcoidosis this year.”


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The FDA’s Orphan Drug designation is granted to medicines intended to treat or prevent rare diseases or disorders that affect fewer than 200,000 individuals.

References

  1. aTyr Pharma announces FDA Orphan Drug designation for ATYR1923 for treatment of sarcoidosis. News release. January 6, 2022. https://www.globenewswire.com/news-release/2022/01/06/2362386/0/en/aTyr-Pharma-Announces-FDA-Orphan-Drug-Designation-for-ATYR1923-for-Treatment-of-Sarcoidosis.html. 
  2. aTyr Pharma announces positive data from phase 1b/2a clinical trial demonstrating consistent dose response for ATYR1923 in pulmonary sarcoidosis. News release. September 13, 2021. https://investors.atyrpharma.com/news-releases/news-release-details/atyr-pharma-announces-positive-data-phase-1b2a-clinical-trial.

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