The FDA has given orphan drug status to CT053, an
investigational chimeric
antigen receptor T-cell therapy for the treatment of multiple
myeloma.
CT053 (CARsgen Therapeutics) is an autologous, fully
human CAR
T-cell therapy that targets the B-cell maturation antigen on the
surface of cancer
cells.
CARsgen’s CT053 is one of the company’s three CAR T-cell
products approved for early-stage clinical trials. The others include humanized
CD19 CAR-T for B-cell
leukemia and lymphoma and
GPC3 CAR-T for hepatocellular
carcinoma and non-small
cell lung cancer.
“FDA orphan designation is an important regulatory
milestone in the continued development and commercialization of
CT053 anti-BCMA CAR-T cells,” Zonghai Li, MD, PhD, founder,
CEO and chief scientific officer of CARsgen said in a press release.
Li added that CT053 showed “outstanding potency” during
an exploratory phase 1 clinical study in China, where 19 of 24 patients with
relapsed or refractory multiple myeloma had a complete response to therapy. In
addition, there were no cases of high-grade (3 or 4) cytokine release syndrome
during the study.
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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