Shanghai, China (Press Release) – CARsgen Thera­peutics Inc., a clin­i­cal-stage bio­pharma­ceu­tical com­pany, today announced the United States Food and Drug Admin­istra­tion (FDA) has granted orphan drug desig­na­tion to its inves­ti­ga­tional CAR T-cell ther­apy fully human anti-BCMA (B Cell Maturation Antigen) au­tol­o­gous chi­meric an­ti­gen re­cep­tor (CAR) T Cells (CT053) for the treat­ment of multiple myeloma.

"FDA orphan desig­na­tion is an im­por­tant regu­la­tory mile­stone in the con­tinued devel­op­ment and com­mer­cial­iza­tion of CT053 anti-BCMA CAR-T cells," said Dr. Zonghai Li, Founder, CEO and CSO of CARsgen. "CT053 has dem­onstrated outstanding potency in an exploratory phase 1 clin­i­cal study in China. A total of 19 of 24 patients with re­lapsed and refractory multiple myeloma showed com­plete response. And im­por­tantly, no event of grade 3 or higher cytokine release syn­drome (CRS) was observed." The CT053 anti-BCMA CAR-T pro­gram has received Inves­ti­ga­tional New Drug (IND) clearance from the US FDA.

Orphan drug desig­na­tion is granted by the FDA Office of Orphan Products Development to pharma­ceu­tical prod­ucts which are in­tended for the treat­ment of rare dis­eases that affect fewer than 200,000 people in the U.S. Under the Orphan Drug Act, the CT053 anti-BCMA prod­uct would be eli­gible for cer­tain benefits in­clud­ing seven years of mar­ket exclusivity in the United States fol­low­ing mar­ket­ing approval by the FDA.

About CARsgen Thera­peutics, Inc.

CARsgen Thera­peutics is a clin­i­cal-stage immune-oncology com­pany committed to the devel­op­ment and com­mer­cial­iza­tion of CAR-T thera­peutics for unmet medical need. The com­pany has col­lab­o­rated with top hos­pi­tals in China to launch several other First-in-Human studies such as anti-GPC3 CAR-T for hepato­cellular carcinoma and squamous lung cancer, anti-EGFR/EGFRvIII CAR-T for glioblastoma multiforme and anti-Claudin18.2 CAR-T for gastric and pancreatic cancer.

For more in­for­ma­tion, please visit: www.carsgen.com.

Source: CARsgen.