The European Commission (EC) has granted orphan-drug designation (ODD) for Astex Pharma’s oral fixed dose ASTX727 to treat Acute Myeloid Leukemia (AML). AML, a blood cancer characterized by the overproduction of immature white blood cells within the bone marrow, is the most common form of leukemia in Europe. ASTX727 is an orally administered combination of the cytidine deaminse (CDA) inhibitor (cedazuridine) and the anti-cancer DNA hypomethylating agent (decitabine) that works to block CDA production in the liver and gut and allow for decitabine to target excess cells within the bone marrow. The EC grants ODD to companies developing medical products that are intended for the treatment, prevention, and diagnosis of life-threatening diseases that affect fewer than five out of 10,000 people in the European Union (EU). Currently, ASTX727 is marketed under the name INQOVI in the U.S. and is approved for the treatment of myelodysplastic syndromes (MDS) and myelomonocytic leukemia (CMML). Astex Pharma, a subsidiary of Otsuka Pharma, is a Japan-based pharmaceuticals company that develops therapies to treat solid tumors and hematological malignancies.
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