FDA Brief: Eprenetapopt Given Fast Track Designation for TP53-Mutant AML
Posted: Tuesday, December 8, 2020
On November 30, the U.S. Food and Drug Administration (FDA) granted Fast Track designation to eprenetapopt for patients with TP53-mutant acute myeloid leukemia (AML). The FDA previously granted Breakthrough Therapy, orphan drug, and Fast Track designations for eprenetapopt for the treatment of patients with TP53-mutant myelodysplastic syndromes (MDS) along with orphan drug designation from the European Medicines Agency for MDS, AML, and ovarian cancer.
Eprenetapopt (also known as APR-246) is a small molecule that has demonstrated reactivation of mutant and inactivated p53 protein by restoring wild-type p53 conformation and function, which induces programmed cell death in human cancer cells. Synergy has previously been reported with chemotherapy and immune-oncology checkpoint inhibitors.
In addition to preclinical testing, a phase I/II clinical trial with eprenetapopt has been completed. Early results have shown a favorable safety profile as well as biologic and confirmed clinical responses in hematologic malignancies and solid tumors with mutations in the TP53 gene.