Bioheng Biotech received Orphan Drug Designation from the U.S. FDA for the treatment of T-ALL.
Nanjing Bioheng Biotech Co., Ltd. Announced 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).
"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.
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.
Bioheng is a clinical-stage biotech company focused on developing novel cellular immunotherapy. The main development pipelines include allogeneic CAR T, which is used for the treatment of hematologic and solid tumors, so as to solve the dilemma of high cost, long time, difficult manufacturing and individualization of traditional CAR T, realize the affordability, accessibility and popularity of cancer treatment, build the bridge of life and health, and let more patients get the cure opportunity. Currently, a high standard GMP-level clinical transformation center has been established to meet the requirements of clinical development and post marketing manufacturing.