CAMBRIDGE (MASSACHUSETTS) (dpa-AFX) - Vertex Pharmaceuticals Inc. (VRTX), Monday announced that its New Drug Submission or NDS for exagamglogene autotemcel has been accepted for Priority Review by Health Canada for the treatment of patients aged 12 years and older with sickle cell disease or SCD and transfusion-dependent beta thalassemia or TDT.
SCD and TDT are debilitating, progressive, life shortening genetic disease, which lead to reduced life expectancy, decreased quality of life and reduced lifetime earnings and productivity.
Vertex explained that exagamglogene autotemcel is a gene-edited cell therapy where a patient's own hematopoietic stem and progenitor cells are edited to increase the production of fetal hemoglobin in red blood cells.
NDS is supported by data from the ongoing Phase 3 studies, CLIMB-111 and CLIMB-121, as well as an ongoing long-term follow-up study, CLIMB-131.
Currently, Vertex's stock is falling 0.19 percent, to $416.83 on the Nasdaq.
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