Tamibarotene granted Fast Track Designation by the U.S. FDA

On April 9, 2024, the U.S. Food and Drug Administration (FDA) granted Fast Track Designation to tamibarotene, an oral selective retinoic acid receptor alpha (RARA) agonist, in combination with azacitidine and venetoclax, for the treatment of patients with newly diagnosed acute myeloid leukemia (AML) with RARA overexpression, as detected by a U.S. FDA approved test, who are aged ≥75 years or ineligible for intensive chemotherapy.¹ Tamibarotene was previously granted Orphan Drug Designation by the U.S. FDA for the treatment of patients with AML.

Tamibarotene is currently being investigated in combination with venetoclax and azacitidine in the phase II SELECT-AML-1 trial (NCT04905407).¹ Results from an interim analysis found that this triplet regimen was associated with a 100% complete remission/complete remission with incomplete count recovery rate vs 70% for venetoclax plus azacitidine alone, with a similar safety profile.¹  Additionally, the ongoing phase III SELECT-MDS-1 trial (NCT04797780) is investigating the combination of tamibarotene and azacitidine for the treatment of patients with newly diagnosed higher-risk myelodysplastic syndromes (HR-MDS) with RARA overexpression.¹ The AML Hub previously reported results from a phase II trial (NCT02807558) assessing tamibarotene in combination with azacitidine for the treatment of patients with newly diagnosed AML.