On June 20, 2021 Nanjing Bioheng Biotech Co., Ltd. reported that UCAR T cell therapy product targeting CD7, code CTD401, received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for the treatment of T-cell acute lymphoblastic leukemia (T-ALL) (Press release, Bioheng Biotech, JUN 20, 2021, View Source [SID1234584179]).
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"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," said Dr. JiangTao Ren, Co-founder and Chief Scientific Officer of Bioheng, "Orphan drug accreditation not only reflects the confidence of the international authority FDA on CTD401, but also is an important milestone of Bioheng gradual globalization."
CTD401 is a universal CAR-T (UCAR-T) cell therapy product developed by Bioheng for the treatment of patients with T-ALL. The product is prepared by collecting peripheral blood mononuclear cells (PBMC) from healthy donors, using a virus encoding a CD7 CAR gene for transduction, and using CRISPR/cas9 technology to knock out key genes.
About ODD
The Orphan Drug Designation program provides orphan status to drugs and biologics which are defined as those intended for the treatment, prevention or diagnosis of a rare disease or condition, which is one that affects less than 200,000 persons in the US or meets cost recovery provisions of the act. After obtaining the ODD, pharmaceutical companies could receive various incentives for the drug development, including 25% federal tax credit for expenses incurred in conducting clinical research within the United States, Waiver of Prescription Drug User Fee Act (PDUFA) fees for orphan drugs, qualifed to compete for research grants from the Office of Orphan Products Development (OOPD) to support clinical studies for orphan drugs, eligibility to receive regulatory assistance and guidance from the FDA in the design of an overall drug development plan and 7-year marketing exclusivity.