Syros Pharmaceuticals, a leader in the development of medicines that control the expression of genes, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to tamibarotene for the treatment of myelodysplastic syndrome (MDS). Tamibarotene, an oral first-in-class selective retinoic acid receptor alpha (RARα) agonist, is currently being evaluated in combination with azacitidine in the SELECT-MDS-1 Phase 3 trial for RARA-positive patients with newly diagnosed higher-risk MDS (HR-MDS).

“The FDA’s orphan drug designation is an important milestone in the development of tamibarotene as a treatment for MDS,” said David A. Roth, M.D., Syros’ Chief Medical Officer. “We believe tamibarotene’s novel mechanism of action, promising clinical activity data, oral delivery, and favorable tolerability profile supports a potential new option for the approximately 30% of HR-MDS patients who are RARA-positive. We are focused on developing the first potential therapy for a targeted population in HR-MDS as we continue to advance our ongoing SELECT-MDS-1 pivotal trial.”

The FDA's Office of Orphan Drug Products grants orphan status to support development of medicines for the treatment of rare diseases that affect fewer than 200,000 people in the United States. Orphan drug designation may provide certain benefits, including a seven-year period of market exclusivity if the drug is approved, tax credits for qualified clinical trials and an exemption from FDA application fees.

The ongoing SELECT-MDS-1 Phase 3 clinical trial is evaluating the safety and efficacy of tamibarotene in combination with azacitidine for RARA-positive patients with newly diagnosed HR-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.

Syros is also evaluating tamibarotene 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. Safety lead-in data from the ongoing SELECT-AML-1 Phase 2 trial is expected in the second half of this year.