Posted by Michael Wonder on 09 Jun 2023
FDA accepts biologics license applications for exagamglogene autotemcel (exa-cel) for severe sickle cell disease and transfusion-dependent beta thalassaemia
8 June 2023 - FDA grants priority review for severe sickle cell disease and standard review for transfusion-dependent beta thalassemia.
Vertex Pharmaceuticals and CRISPR Therapeutics today announced that the US FDA has accepted the biologics license applications for the investigational treatment exagamglogene autotemcel (exa-cel) for severe sickle cell disease and transfusion-dependent beta thalassaemia.
