FDA Grants Orphan Drug Designation to ATYR1923 for Sarcoidosis Treatment

Jul 18 2025

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FDA Grants Orphan Drug Designation to ATYR1923 for Sarcoidosis Treatment

aTyr Pharma, Inc., a clinical-stage biotherapeutics company focused on the discovery and development of novel medicines based on novel biological pathways, announced today that the U.S. Food and Drug Administration (FDA) has granted the company orphan drug designation for ATYR1923, its lead therapeutic candidate for the treatment of Sarcoidosis. ATYR1923 is a potential first-in-class immunomodulator that suppresses innate and adaptive immune responses in uncontrolled inflammatory disease states.

Source: aTyr Pharma, Inc.

Sarcoidosis is a systemic inflammatory disease characterized by granuloma formation and scarring, which in some cases, permanently impairs organ function. In people with sarcoidosis, abnormal masses or nodules called granulomas, consisting of inflamed tissues, form in specific body organs. These granulomas may alter the standard structure and possibly the function of the affected organs. Sarcoidosis symptoms and signs differ depending on which organs are affected. It can develop gradually and cause symptoms to last for years. Symptoms may appear suddenly and then vanish just as quickly. The goals of sarcoidosis management are to prevent or control organ damage, relieve symptoms, and improve the patient’s quality of life. An evaluation by a pulmonologist is strongly recommended. For patients with extrapulmonary involvement, a multidisciplinary approach may be required. The patient may need to see an ophthalmologist for ocular disease, a cardiologist for cardiac disease, a neurologist for neurological disease, a nephrologist for renal disease, etc.

  • The annual incidence of Sarcoidosis varies between 8 to 15 cases per 100,000 population in the USA.

However, the current Sarcoidosis treatment market share, market uptake, attribute analysis concerning the most potential emerging therapies (ATYR1923, CMK389, ARA 290, Selexipag, etc.) have been provided under the market outlook section of the study covering 8 MM countries; The United States, EU5 (Germany, Spain, France, Italy, UK) Japan and China.

In terms of pharmacologic therapies, several pharmaceutical products are being approved and under different phases of development for Sarcoidosis treatment. The key companies in the advanced development stage are (Novartis Pharmaceuticals, Araim Pharmaceuticals, Inc., Actelion, Gilead Sciences, etc.)

Based on solid domain and business knowledge, Thelansis Knowledge Partners has published the market outlook report on Sarcoidosis to provide a clear understanding of disease area background, epidemiology, current and future competitions, the country-specific standard of care, and the complete market forecast for 2020 to 2030.

Thelansis specializes in pharmaceutical market research and market Insight Report Company, published reports across the therapeutic area, including rare / ultra-rare and mainstream indication. Over the period, we have built a robust repository of 6,000+ Bio-pharma reports that cover Epidemiology studies and Market forecasting based on the KOL opinions.

Competitive intelligence and track of trial results throughout the phases of development executed by a team of a mix of Scientific and Business backgrounds. As an organization, the primary focus is to provide real-world data evidence and market insight to pharmaceutical companies for their decision-making.

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