On August 3, 2022 Syros Pharmaceuticals (NASDAQ:SYRS), a leader in the development of medicines that control the expression of genes, reported that the European Medicines Agency (EMA) issued a positive opinion on the Company’s application for orphan drug designation for tamibarotene for the treatment of myelodysplastic syndrome (MDS) (Press release, Syros Pharmaceuticals, AUG 3, 2022, View Source [SID1234617423]). 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).
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Previously, the U.S. Food and Drug Administration (FDA) granted orphan drug designation to tamibarotene in MDS in February 2022.
"We are pleased that the EMA has issued a positive opinion for orphan drug designation for tamibarotene as it represents an important milestone for MDS patients, who have an urgent need for effective, tolerable, and convenient treatment options," said David A. Roth, M.D., Syros’ Chief Medical Officer. "We believe tamibarotene has the potential to change the current standard of care and become the first therapy for a targeted population in HR-MDS. We continue to advance our SELECT-MDS-1 trial and are looking forward to announcing pivotal data in late 2023 or early 2024."
Orphan drug designation in the European Union (EU) is granted by the European Commission based on a positive opinion issued by the EMA Committee for Orphan Medicinal Products. The EMA’s orphan designation is available to companies developing treatments for life-threatening or chronically debilitating conditions that affect fewer than five in 10,000 persons in the EU. Medicines that meet the EMA’s orphan designation criteria qualify for financial and regulatory incentives that include a 10-year period of marketing exclusivity in the EU after product approval, protocol assistance from the EMA at reduced fees during the product development phase and access to centralized marketing authorization.
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 from both the FDA and EMA. Data from the safety lead-in portion of the SELECT-AML-1 Phase 2 trial is expected in the second half of this year.