Revolution in sickle cell disease treatment on the way in Europe

Genetic disease company Vertex Pharmaceuticals (Nasdaq: VRTX) has secured conditional marketing authorization for Casgevy (exagamglogene autotemcel) in Europe.

Also known as exa-cel, the product is a CRISPR/Cas9 gene-edited therapy for people with severe sickle cell disease (SCD).

Contingent on further data confirming benefit, the European Commission has authorized marketing of the product for certain patients over the age of 12, where a matched stem cell donor cannot be found.