European Medicines Agency Issues Positive Opinion on Orphan Drug Designation for Tamibarotene for the Treatment of MDS

The European Medicines Agency (EMA) issued a positive opinion on the Orphan Drug Designation for tamibarotene, an oral first-in-class selective retinoic acid receptor alpha (RARA) agonist, for the treatment of myelodysplastic syndrome (MDS).

The U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to tamibarotene for the treatment of MDS in February 2022.

Tamibarotene is currently being evaluated in combination with azacitidine in the SELECT-MDS-1 phase III trial for RARA-positive patients with newly diagnosed higher-risk MDS. Data from the pivotal trial are expected in the fourth quarter of 2023 or the first quarter of 2024, with a potential new drug application filing expected in 2024.

Tamibarotene is also being evaluated in combination with azacitidine and venetoclax for RARA-positive patients with newly diagnosed unfit acute myeloid leukemia (AML), for which tamibarotene had previously received Orphan Drug Designation from both the FDA and EMA.

Source: Business Wire via Syros Pharmaceuticals, August 2022