On December 22, 2021 Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported that the European Commission has granted Orphan Drug Designation to SNDX-5613, the Company’s highly selective oral menin inhibitor, for the treatment of acute myeloid leukemia (AML) (Press release, Syndax, DEC 22, 2021, View Source [SID1234597607]).
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"Supported by a growing body of clinical data, we firmly believe that SNDX-5613 is ideally positioned to serve as a best-in-class, meaningful intervention for patients with NPM1 and MLLr acute leukemias," said Briggs W. Morrison, M.D., Chief Executive Officer of Syndax. "Receipt of Orphan Drug Designation from the European Commission further validates the important role that SNDX-5613 could play in the treatment of this highly underserved patient population, and we are fully committed to ensuring that it is able to reach as many of these patients as possible."
The European Commission grants Orphan Drug Designation for medicinal products intended to treat life-threatening or chronically debilitating conditions that affect fewer than five in 10,000 people in the European Union (EU) and when no satisfactory method of diagnosis, prevention, or treatment of the condition can be authorized. The designation provides certain benefits and incentives in the EU, including protocol assistance, fee reductions, and ten years of market exclusivity once the medicine is on the market.
SNDX-5613 was previously granted Orphan Drug Designation for the treatment of adult and pediatric AML by the U.S. Food and Drug Administration.
About SNDX-5613
SNDX-5613 is a potent, selective, small molecule inhibitor of the menin-MLL binding interaction that is being developed for the treatment of mixed lineage leukemia rearranged (MLLr) acute leukemias including acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML), and NPM1 mutant AML. In preclinical models of MLLr acute leukemias, SNDX-5613 demonstrated robust, dose-dependent inhibition of tumor growth, resulting in a marked survival benefit. Menin-MLL interaction inhibitors have also demonstrated robust treatment benefit in multiple preclinical models of NPM1 mutant AML, which represents the most frequent genetic abnormality in adult AML. SNDX-5613 is currently being evaluated in the Company’s AUGMENT-101 Phase 1/2 open-label clinical trial for the treatment of relapsed/refractory (R/R) acute leukemias. SNDX-5613 was granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) and European Commission for the treatment of patients with AML, and Fast Track designation by the U.S. FDA for the treatment of adult and pediatric patients with R/R acute leukemias harboring a mixed lineage leukemia rearranged MLLr or NPM1 mutation.