FDA Grants Orphan Drug Designation to Enzomenib for Acute Lymphoblastic Leukaemia
Friday, July 31, 2026
Sumitomo Pharma America has announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to enzomenib (DSP-5336) for the treatment of patients with acute lymphoblastic leukaemia (ALL).
Enzomenib is an investigational oral small-molecule inhibitor that targets the interaction between menin and lysine-specific methyltransferase 2A (KMT2A), a pathway involved in the development and growth of acute leukaemia and other cancers.
The designation follows the FDA’s earlier Orphan Drug Designation for enzomenib in acute myeloid leukaemia (AML), which was granted in 2022. The therapy is currently being evaluated in a Phase I/II dose-escalation and dose-expansion study involving patients with relapsed or refractory acute leukaemia, as well as in the registrational Phase II Horizen-1 trial for patients with relapsed or refractory AML or ALL carrying KMT2A rearrangements or NPM1 mutations.
Acute lymphoblastic leukaemia is a fast-progressing blood cancer that develops when the bone marrow produces excessive immature lymphocytes. If left untreated, the disease can lead to reduced blood cell production, increasing the risk of infections, anaemia and bleeding, and may spread to the brain and spinal cord.
Preclinical studies have shown that enzomenib selectively inhibits the growth of acute leukaemia cells with KMT2A rearrangements or NPM1 mutations. The therapy has also demonstrated the ability to reduce the expression of leukaemia-associated genes while promoting markers linked to cell differentiation.
In addition to its orphan designation for AML, enzomenib received FDA Fast Track Designation in 2024 for the treatment of relapsed or refractory AML with KMT2A rearrangements or NPM1 mutations. Japan’s Ministry of Health, Labour and Welfare also granted Orphan Drug Designation for the same indication in 2024.
Source: businesswire.com
