FDA grants orphan drug designation to natural killer cell therapy for multiple myeloma
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The FDA granted orphan drug designation to CellProtect, a modified natural killer cell therapy for the treatment of patients with multiple myeloma, according to the agent’s manufacturer.
CellProtect (XNK Therapeutics) is an investigational autologous cell therapy produced through ex vivo expansion and activation of natural killer cells from peripheral blood of patients with cancer.
The technology used to produce the therapy is based on natural killer cell research performed at Karolinska Institute in Stockholm. The manufacturing process takes a little less than 3 weeks, according to the manufacturer.
“Obtaining an [orphan drug designation] by the FDA is a significant milestone for XNK and our goal of taking [this] drug candidate to the next level,” Johan Liwing, CEO of XNK Therapeutics, said in a press release. “This is the starting point for us to expand clinical development into the most important market globally for cancer treatment.”
Researchers tested CellProtect in the phase 1/phase 2 ACP-001 trial, results of which were presented at this year’s European Hematology Association Annual Meeting. The trial evaluated CellProtect as consolidation treatment after high-dose stem cell transplantation among patients with newly diagnosed multiple myeloma.
Safety and tolerability served as the primary objective.
According to XNK Therapeutics, the safety analysis showed no serious adverse events and the efficacy results showed an OS rate of 100% after median follow-up of 60 months. Median PFS was 34 months.
XNK intends to start a phase 2 trial of CellProtect for patients with multiple myeloma “in the near future,” according to the press 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.
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