Aspacytarabine granted orphan drug designation by the FDA

On August 1, 2022, aspacytarabine, an investigational novel antimetabolite, was granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of patients with myelodysplastic syndromes (MDS).¹

Aspacytarabine (BST-236) is a novel proprietary antimetabolite. It is made up of cytarabine covalently bound to asparagine. Aspacytarabine enables high-dose therapy with lower systemic exposure to free cytarabine and relative sparing of normal tissues, making it a suitable therapy for older adults who are unfit for intensive therapy. The FDA previously granted aspacytarabine orphan drug designation for the treatment of patients with acute myeloid leukemia (AML) in 2019.¹

The safety and efficacy of aspacytarabine as a single-agent first-line therapy for patients with AML have previously been demonstrated, and additional studies are ongoing to assess aspacytarabine as a second-line treatment for patients with relapsed/refractory MDS. The AML Hub has previously published an article and an expert opinion on aspacytarabine.