FDA grants orphan drug designation to natural killer cell therapy for GI cancers
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The FDA granted orphan drug designation to CYNK-101, an investigational natural killer cell-based therapy for treatment of advanced HER2/neu-positive gastric or gastroesophageal junction adenocarcinoma.
CYNK-101 (Celularity) is a gene-edited, allogeneic natural killer (NK) cell therapy composed of human placental hematopoietic stem cells engineered to express a high-affinity and cleavage-resistant CD16 (FCGRIIIA) variant.
The FDA previously granted fast track designation to CYNK-101 for the same indication.
A nonrandomized phase 1/phase 2A clinical trial is evaluating CYNK-101 in combination with standard chemotherapy, trastuzumab (Herceptin, Genentech) and pembrolizumab (Keytruda, Merck) as first-line therapy for patients with locally advanced unresectable or metastatic HER2/neu-positive gastric or gastroesophageal junction adenocarcinoma.
“This [orphan drug] designation underscores the significant unmet need for these patients and CYNK-101’s potential in a new first-line treatment strategy,” Robert J. Hariri, MD, PhD, chairman and CEO of Celularity, said in a company-issued press release. “We are committed to forging new treatment strategies that leverage the unique properties of placental-derived cellular therapies to improve the lives of patients with this difficult-to-treat cancer.”
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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