Policy & Regulation
Syros Receives Positive Opinion on Orphan Drug Designation from the European Medicines Agency for Tamibarotene for the Treatment of MDS
4 August 2022 - - The European Medicines Agency issued a positive opinion on US-based Syros Pharmaceuticals' (NASDAQ: SYRS) application for orphan drug designation for tamibarotene for the treatment of myelodysplastic syndrome.

Tamibarotene, an oral first-in-class selective retinoic acid receptor alpha 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).

Previously, the US Food and Drug Administration granted orphan drug designation to tamibarotene in MDS in February 2022.

Orphan drug designation in the European Union 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 4Q23 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, 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.

Syros is redefining the power of small molecules to control the expression of genes. Based on its unique ability to elucidate regulatory regions of the genome, Syros aims to develop medicines that provide a profound benefit for patients with diseases that have eluded other genomics-based approaches.

Syros is advancing a robust clinical-stage pipeline, including: tamibarotene, a first-in-class oral selective RARα agonist in RARA-positive patients with higher-risk myelodysplastic syndrome and acute myeloid leukemia; SY-2101, a novel oral form of arsenic trioxide in patients with acute promyelocytic leukemia; and SY-5609, a highly selective and potent oral CDK7 inhibitor in patients with select solid tumors.

Syros also has multiple preclinical and discovery programs in oncology and monogenic diseases.