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FDA grants fast track, orphan drug designations to APR-246 for myelodysplastic syndrome
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The FDA granted fast track designation to APR-246 for the treatment of patients with myelodysplastic syndrome who have TP53 mutations.
The agency also granted orphan drug designation to APR-246 (Aprea Therapeutics) for treatment of myelodysplastic syndrome.
These designations underscore the “significant unmet need in this disease,” Christian S. Schade, president and CEO of Aprea, said in a company-issued press release.
“With our phase 3 clinical study in myelodysplastic syndrome underway, we look forward to continuing our productive dialogue with FDA and bringing APR-246 to patients as soon as possible,” Schade said.
The p53 tumor suppressor gene is the most commonly mutated gene in cancer among humans, occurring in approximately half of all tumors. The mutations frequently are associated with resistance to cancer therapies and poor OS.
Research has shown APR-246 can reactivate mutant and inactivated p53 protein by restoring wild-type p53 conformation and function, according to the press release. This helps induce programmed cell death in human cancer cells.
The agent has demonstrated preclinical antitumor activity in several hematologic malignancies and solid tumors, including myelodysplastic syndrome, acute myeloid leukemia and ovarian cancer.
Fast track designation helps to expedite drug development, review and potential approval.
The FDA 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. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.