Vertex Pharmaceuticals Inc. revealed on Monday that its New Drug Submission (NDS) for the medication exagamglogene autotemcel (eax-gam-log-en auto-tem-sel) has gained Priority Review status from Health Canada. This drug is aimed at treating patients aged 12 years and above suffering from sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).
These are significant and escalating genetic diseases that affect life expectancy, quality of life, and economic productivity by reducing both lifetime earnings and overall productivity.
Exagamglogene autotemcel is a groundbreaking cell therapy that uses gene-editing technology on patient's own hematopoietic stem and progenitor cells to boost the production of fetal hemoglobin in red blood cells.
The support for the NDS has been provided by data from continuous Phase 3 studies, notably CLIMB-111 and CLIMB-121, in addition to an ongoing long-term follow-up study, CLIMB-131.
Presently, shares in Vertex are experiencing a slight decrease of 0.19 percent, dropping to $416.83 on the Nasdaq.
