FDA grants orphan drug designation to T-cell therapies for advanced liver cancer
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The FDA granted orphan drug designation to two investigational T-cell immunotherapies for the treatment of advanced hepatocellular carcinoma.
ET140203 (Eureka Therapeutics) and ECT204 (Eureka Therapeutics) are autologous, gene-edited T-cell therapies.
ET140203 is engineered to express a T-cell receptor (TCR) that targets an alpha fetoprotein-peptide/HLA-A2 complex. ECT204 is engineered to express a TCR that targets the glypican 3 protein. Both proteins are commonly expressed on the surface of liver cancer cells.
Both therapies have been developed using Eureka’s proprietary ARTEMIS cell technology platform, designed to produce treatments that prevent T-cell hyperactivity and subsequent cytokine release syndrome, according to the manufacturer.
“These designations for Eureka’s clinical candidates underscore the significant unmet medical need for more effective liver cancer treatment options,” Cheng Liu, PhD, president and CEO of Eureka Therapeutics, said in a company-issued press release. “We are committed to working closely with regulators, clinical investigators, patients and their families to advance these programs in the clinic.”
Eureka is enrolling patients in multicenter phase 1/phase 2 clinical trials to assess the safety and efficacy of both cell therapies.
The ARYA-1 trial is designed to evaluate ET140203 for adults with advanced HCC. The ARYA-2 study is designed to evaluate the therapy for patients aged 21 years and younger with relapsed or refractory hepatoblastoma, hepatocellular neoplasia or HCC.
The ARYA-3 study will evaluate ECT204 for adults with glypican 3-positive advanced HCC.
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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