FDA grants orphan drug designation to NKX101 for acute myeloid leukemia
The FDA granted orphan drug designation to NKX101 for treatment of acute myeloid leukemia.
NKX101 (Nkarta) uses natural killer cells derived from peripheral blood of healthy donors and engineered with membrane-bound interleukin-15 and a chimeric antigen receptor that targets NKG2D ligands on tumor cells, according to a Nkarta-issued press release.
Engineering NKX101 with the proprietary NKG2D-based CAR significantly increased the ability of natural killer cells to recognize and kill tumor cells, according to preclinical models.
A phase 1 trial is underway to assess NKX101 for adults with relapsed or refractory AML or myelodysplastic syndrome. Preliminary results are expected in the first half of 2022.
“This orphan drug designation acknowledges the urgent need for new treatment options for patients with AML,” Kanya Rajangam, MD, PhD, chief medical officer of Nkarta, said in the release.
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.