Posted by Michael Wonder on 03 Apr 2024
Vertex announces new drug submission for exagamglogene autotemcel (exa-cel) has been accepted for priority review by Health Canada for the treatment of sickle cell disease and transfusion-dependent beta thalassaemia
1 April 2024 - Exa-cel is the first CRISPR-based gene-edited therapy to be submitted for Health Canada review.
Vertex Pharmaceuticals today announced its new drug submission for exagamglogene autotemcel (exa-cel) has been accepted for Priority Review by Health Canada for the treatment of patients aged 12 years and older with sickle cell disease with recurrent vaso-occlusive crises and for the treatment of patients aged 12 years and older with transfusion-dependent beta thalassaemia.
