Vertex and CRISPR Therapeutics have taken a small step toward a landmark approval, securing European Medicines Agency (EMA) validation of the approval application for their CRISPR-Cas9 candidate.
The candidate, exagamglogene autotemcel (exa-cel), is an autologous cell therapy treatment for sickle cell disease and transfusion-dependent beta thalassemia. By using CRISPR-Cas9 to edit a patient’s own hematopoietic stem cells, the partners modify cells to produce high levels of fetal hemoglobin. One-time treatment with exa-cel has freed patients from transfusions and vaso-occlusive crises.
In September, Vertex and CRISPR outlined plans to file the data needed to support approval in Europe by the end of 2022. Now, the EMA has validated the application, confirming that all of the elements needed for its scientific assessment are present.
Exa-cel, formerly known as CTX001, has PRIME designation from the EMA, making it eligible for a priority review that usually takes 150 days, rather than the typical 210 days. The timeline suggests exa-cel could win approval in Europe later this year and become the first marketed CRISPR-Cas9 treatment.
Yet, history shows approval could be just the start of the challenges facing Vertex and CRISPR in Europe. The companies are following in the footsteps of bluebird bio, which won conditional EMA approval for its autologous lentiviral gene therapy in beta thalassemia in 2019, only to quit the market two years later after concluding that “there is an element of the European system that’s frankly broken.”
Bluebird’s retreat gives Vertex and CRISPR a clear run at the market opportunity for a potentially curative treatment of beta thalassemia, which is more common in Europe than the U.S., and sickle cell. But it also shows how Europe’s fragmented payer landscape can be a hostile environment to expensive, advanced therapies.
Vertex and CRISPR are advancing toward an EMA decision in Europe while working to bring exa-cel to the U.S. market. The partners expect to complete a rolling submission to the FDA by the end of the first quarter.