The Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for pracinostat (MEI Pharma) in combination with azacitidine for the treatment of acute myeloid leukemia (AML) in newly diagnosed patients who are ≥75 years old or ineligible for intensive chemotherapy.

The FDA designation was based on data from a Phase 2 study evaluating pracinostat plus azacitidine in 50 elderly patients with newly diagnosed AML ineligible for induction chemotherapy. Results from the study showed that patients treated with the combination therapy had a median overall survival of 19.1 months and a complete response (CR) rate of 42%. No unexpected toxicities were observed, with the most common grade 3/4 treatment-emergent adverse events presenting as febrile neutropenia, thrombocytopenia, anemia and fatigue. 

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