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FDA grants orphan drug designation to enzomenib for ALL
On July 30, 2026, the U.S. Food and Drug Administration (FDA) granted orphan drug designation to enzomenib, an oral menin inhibitor, for the treatment of patients with acute lymphoblastic leukemia (ALL).1
Enzomenib is currently being assessed in the ongoing phase I/II Horizen-1 trial (NCT04988555) as monotherapy or in combination with venetoclax + azacitidine, gilteritinib, or daunorubicin + cytarabine (7+3) for the treatment of relapsed/refractory (R/R) acute leukemias, including those with KMT2A rearrangements (KMT2Ar) or NPM1 mutations (NPM1m).1,2 Preliminary results (as of January 2024) demonstrated an overall response of 45%, with a complete response (CR) plus CR with partial hematologic recovery (CRh) rate of 23% in all patients with KMT2Ar or NPM1m.3 No dose-limiting toxicities have been reported and the most common adverse events included vomiting (15.5%) and nausea (12.1%).3 QT prolongation was reported in one patient, and was managed with dose interruption and reduction.3
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