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Eprenetapopt receives FDA Fast Track designation for the treatment of AML

Dec 1, 2020

On November 30, 2020, it was announced that the U.S. Food and Drug Administration (FDA) granted Fast Track designation to eprenetapopt/APR-246 for the treatment of patients with TP53-mutated acute myeloid leukemia (AML).1

Eprenetapopt is a small molecule that reactivates mutant and inactivated p53 protein to induce apoptosis in cancer cells. It has previously received FDA Breakthrough Therapy designation, orphan drug designation, and Fast Track designation for the treatment of myelodysplastic syndromes (MDS), as well as orphan drug designation from the European Medicines Agency (EMA) for MDS, AML, and ovarian cancer.

The emerging clinical data are encouraging, as shown by the safety and efficacy results presented at this year’s European Hematology Association (EHA) Annual Congress, and clinical trials evaluating combination therapies with azacitidine or azacitidine plus venetoclax are continuing to expand to identify the best treatment regimen.

  1. Aprea Therapeutics. Aprea Therapeutics receives FDA Fast Track designation for eprenetapopt in the treatment of TP53 mutant acute myeloid leukemia (AML). Published Nov 30, 2020. Accessed Dec 1, 2020.