FDA Grants Fast Track Designation to Tamibarotene for Higher-Risk MDS

The U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to tamibarotene, an oral first-in-class selective retinoic acid receptor alpha (RARα) agonist, for treatment of higher-risk myelodysplastic syndromes (MDS).

The ongoing phase III SELECT MDS-1 trial is evaluating a combination of tamibarotene plus azacitidine in newly diagnosed higher-risk patients with MDS who have RARA overexpression. Data from the trial is expected in the third quarter of 2024.

The selective RARα agonist is also under evaluation as part of a triplet combination with venetoclax and azacitidine in patients with newly diagnosed acute myeloid leukemia (AML) who are unfit and have RARA overexpression. Initial data from the randomized portion of the SELECT-AML-1 phase II trial is expected in the fourth quarter of 2023, with additional data expected in 2024.

Source: Syros Pharmaceuticals, January 2023