Posted by Michael Wonder on 03 Apr 2023
Vertex and CRISPR Therapeutics complete submission of rolling biologics license applications to the US FDA for exa-cel for the treatment of sickle cell disease and transfusion-dependent beta thalassaemia
3 April 2023 - EU and UK filings completed in 2022; submissions validated by EMA and MHRA and the review procedure has begun as of January 2023.
Vertex Pharmaceuticals and CRISPR Therapeutics today announced the completion of the rolling biologics license applications to the US FDA for the investigational treatment exagamglogene autotemcel (exa-cel) for sickle cell disease and transfusion-dependent beta thalassaemia.
