FDA grants orphan drug designation to CAR-T for mesothelioma
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The FDA granted orphan drug designation to SynKIR-110, an investigational chimeric antigen receptor T-cell therapy, for treatment of mesothelin-expressing mesothelioma.
SynKIR-110 (Verismo Therapeutics) is a gene-edited CAR T-cell therapy with next-generation dual-chain signaling.
The agent is based on Versimo’s proprietary KIR-CAR platform, which uses a modified natural killer (NK) cell-like receptor to improve T-cell persistence and efficacy against solid tumors.
"Mesothelioma is a rare and deadly disease for which few other treatment options currently exist,” Bryan Kim, DMD, CEO of Verismo Therapeutics, said in a company-issued press release. “We look forward to working with the mesothelioma community to advance SynKIR-110 as a potential treatment while we continue to expand clinical investigation of this novel platform in other cancers in the solid tumor space."
The FDA based the orphan drug designation on preclinical studies that showed SynKIR-110 could produce complete remissions in mouse models of glioblastoma and mesothelioma.
Verismo Therapeutics plans the phase 1 STAR-101 trial, a first-in-human study to evaluate the safety and efficacy of SynKIR-110 for patients with mesothelin-expressing ovarian cancer, mesothelioma and cholangiocarcinoma. The company expects to begin enrolling patients in the first quarter of 2023 at the initial trial site, Hospital of the University of Pennsylvania.
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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