Tamibarotene granted fast track designation by the FDA

On January 26, 2023, tamibarotene, a first-in-class oral selective retinoic acid receptor alpha (RARα) agonist, was granted fast track designation by the U.S. Food and Drug Administration (FDA) for the treatment of higher-risk myelodysplastic syndromes (MDS).¹

Tamibarotene is currently being examined in combination with azacitidine in the international multicenter SELECT-MDS-1 phase III randomized placebo-controlled trial for patients with newly diagnosed, higher-risk MDS with RARα gene overexpression.¹ Patients with higher-risk MDS, up to 30% of whom overexpress RARα,¹ have a poor prognosis, with around half progressing to acute myeloid leukemia.² In the trial, patients will be randomly assigned to receive azacitidine plus tamibarotene or placebo in a ratio of 2:1.²

Tamibarotene was granted orphan drug designation in MDS by the U.S. FDA on February 2, 2022,³ and is currently also being investigated in combination with venetoclax plus azacitidine in unfit patients with newly diagnosed acute myeloid leukemia and RARα overexpression in the SELECT-AML-1 phase II trial.¹