Business
aTyr Pharma Announces FDA Orphan Drug Designation for ATYR1923 for Treatment of Sarcoidosis
Company expects to initiate registrational trial of ATYR1923 in pulmonary sarcoidosis in 2022 SAN DIEGO, Jan. 06, 2022 (GLOBE NEWSWIRE) -- aTyr Pharma, Inc.
About this update from Atyr Pharma, Inc.
[{"type":"text","content":"Company expects to initiate registrational trial of ATYR1923 in pulmonary sarcoidosis in 2022\nSAN DIEGO, Jan. 06, 2022 (GLOBE NEWSWIRE) -- aTyr Pharma, Inc. (Nasdaq: LIFE), a clinical stage biotherapeutics company engaged in the discovery and development of innovative medicines based on novel biological pathways, today announced that the U.S. Food and Drug Administration (FDA) has granted the company orphan drug designation for its lead therapeutic candidate, ATYR1923, for the treatment of sarcoidosis. ATYR1923 is a potential first-in-class immunomodulator that downregulates innate and adaptive immune responses in uncontrolled inflammatory disease states. Clinical proof-of-concept was recently established for ATYR1923 in a Phase 1b/2a study in patients with pulmonary sarcoidosis, the main form of the disease, and the company expects to initiate a registrational trial in this indication this year. The FDA’s Office of Orphan Drug Products grants orphan status to support the development of medicines for patients with unmet needs for rare disorders affecting fewer than 200,000 people in the United States. Orphan drug designation provides certain benefits, including the potential for seven years of market exclusivity following regulatory approval, exemption from FDA application fees and tax credits for qualified clinical trials. “The receipt of orphan drug designation for ATYR1923 for sarcoidosis is an important milestone and recognizes the unmet need for the close to 200,000 patients living with this chronic, debilitating disease,” said Sanjay S. Shukla, M.D., M.S., President and CEO of aTyr. “Current treatment options for patients with pulmonary sarcoidosis are limited. 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. We look forward to initiating a registrational trial in pulmonary sarcoidosis this year.” Sarcoidosis is an inflammatory disease characterized by the formulation of granulomas, clumps of inflammatory cells, in one or more organs of the body. The lungs are affected in >90% of cases. Almost 200,000 Americans live with sarcoidosis and the prognosis ranges from benign and self-limiting to chronic, debilitating disease, in some cases with scarring of t...