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# Orphan Status for Syros Pharma’s Leukemia Therapy
- URL: https://www.fdaweb.com/orphan-status-for-syros-pharmas-leukemia-therapy/
- Published: 2017-08-21T12:00:00.000Z
- Updated: 2026-09-14T22:42:15.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5139429

FDA has granted Syros Pharmaceuticals an orphan drug designation for SY-1425 for treating acute myeloid leukemia (AML). SY-1425 is described by the company as an oral first-in-class selective retinoic acid receptor alpha agonist. It is currently in a Phase 2 clinical trial in genomically defined subsets of patients with AML and myelodysplastic syndrome (MDS).  

“In preclinical studies, SY-1425 promoted differentiation of AML cells with high *RARA* or *IRF8* expression and inhibited tumor growth and prolonged survival in patient-derived xenograft models of AML with high *RARA* expression,” the company says. It estimates that about one-third of AML and MDS patients have either the *RARA* or *IRF8* biomarker, or both.