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FDA grants orphan drug designation for APTO-253 for AML
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The FDA granted orphan drug designation to APTO-253 for the treatment of patients with acute myeloid leukemia, according to a press release from the drug’s manufacturer.
APTO-253 (Aptose Biosciences) — a first-in-class inducer of the Krüppel-like factor 4 (KLF4) gene — is currently under evaluation in a phase 1b trial of patients with AML, high-risk myelodysplastic syndrome and other hematologic malignancies.
Epigenetic suppression of the KLF4 gene acts as a key transforming event in AML, according to the press release. APTO-253 exhibited a positive safety profile without suppressing normal bone marrow in patients with AML.
Preclinical studies have showed APTO-253 has strong single-agent activity to kill AML cells and robust synergy in combination with other agents, according to the release.
“AML is a particularly challenging cancer of the blood and bone marrow for which there are currently few treatment options,” William G. Rice, PhD, chairman, president and CEO of Aptose Biosciences, said in a press release. “APTO-253, with its unique mechanism of action, has the potential to emerge as an entirely new therapeutic agent for this patient population, and receiving orphan drug designation is a key regulatory milestone along the path.”
The FDA’s Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States.