FDA grants orphan drug designation to cell therapy for T-cell ALL
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The FDA granted orphan drug designation to CTD401, a chimeric antigen receptor T-cell therapy for the treatment of T-cell acute lymphoblastic leukemia, according to the agent’s manufacturer.
CTD401 (Bioheng Biotech) is an allogeneic, gene-edited CAR T-cell therapy that targets the CD7 protein on the surface of cancer cells. The agent is derived from peripheral blood mononuclear cells from healthy donors and uses CRISPR/Cas-9 technology to knock out genes with the aim of preventing CAR T-cell fratricide or T-cell aplasia that can occur when using a T-cell lineage antigen such as CD7.
“CTD401 is the first innovative product based on our next-generation generic CAR-T technology platform. It has excellent efficacy and superior safety in exploratory clinical studies,” JiangTao Ren, PhD, co-founder and chief scientific officer of Bioheng, said in a company-issued press release. “Orphan drug accreditation not only reflects the confidence of the international authority FDA [in] CTD401, but also is an important milestone of [Bioheng’s] gradual globalization.”
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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