FDA grants orphan drug designation to CAR-T for acute myeloid leukemia
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The FDA granted orphan drug designation to KITE-222, an investigational chimeric antigen receptor T-cell therapy, for treatment of adults with relapsed or refractory acute myeloid leukemia.
KITE-222 (Kite Pharma/Gilead Sciences) — an autologous, gene-edited CAR T-cell therapy — targets C-type lectin-like molecule-1 (CLL-1), which is expressed by lineage-committed myeloid cells and absent on healthy hematopoietic stem cells.
“The FDA’s decision to grant this designation to KITE-222 reflects the urgent need to develop novel therapeutics for people living with AML, a devastating disease with poor prognosis,” Frank Neumann, MD, PhD, Kite’s global head of clinical development, said in a company-issued press release. “We look forward to its continued investigation and sharing results with the scientific community.”
Kite is enrolling patients in a multicenter phase 1 study designed to evaluate the safety and feasibility of KITE-222 for adults with relapsed or refractory AML.
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.
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