Vertex-CRISPR’s Casgevy Gets Positive EMA Panel Opinion, Approval Decision in Q1 2024

EMA building_iStock, Lubo Ivanko

Pictured: European Medicines Agency sign on building/iStock, Lubo Ivanko

Following in the footsteps of U.K. and U.S. regulators, a European Medicines Agency committee on Thursday endorsed Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy, which is intended for the treatment of sickle cell disease and transfusion‑dependent beta thalassemia in Europe. 

EMA’s Committee for Medicinal Products for Human Use “adopted a positive opinion, recommending the granting of a conditional marketing authorization” for Casgevy, according to Thursday’s announcement.  

The European Commission is expected to make a final decision in February 2024, according to Vertex’s Friday announcement regarding CHMP’s positive opinion. Historically, approval decisions have nearly always followed the committee’s recommendations. 

Casgevy was approved by the FDA on Dec. 8 for the treatment of sickle cell disease (SCD). The gene therapy utilizes Jennifer Doudna’s Nobel Prize-winning CRISPR/Cas9 technology. 

However, an EMA approval will not necessarily make Casgevy readily available for patients in Europe. Gene therapies are complex and expensive. In the U.S., Casgevy’s list price is $2.2 million per patient, which is greater than the non-elderly lifetime burden of total medical costs for SCD estimated at around $1.7 million

The high cost is cause for concern for European patients. After bluebird bio’s gene therapy for transfusion-dependent beta thalassemia (TDT) was approved in 2019, the company ended up pulling out of the European market in 2021 due to government negotiators lowballing on the therapy’s pricing. At the time, bluebird called the environment “untenable” for gene therapies. 

“There is an urgent need for new potentially curative treatments in beta thalassemia and sickle cell disease, as people with these diseases still have a shorter life expectancy than the general population and an impaired quality of life,” Franco Locatelli, a principal investigator in the CLIMB-111 and CLIMB-121 studies, said in a statement in Vertex’s Friday announcement. 

In Europe, the mean age of death for SCD patients is around 40 years old, according to Vertex, with TDT life expectancy around the same. While stem cell transplantation can offer a curative option for both diseases, it’s only an option for a small fraction of patients due to lack of available matched donors. 

The FDA is expected to make a decision on Casgevy’s approval for TDT by the end of March 2024. 

Kate Goodwin is a freelance life science writer based in Des Moines, Iowa. She can be reached at kate.goodwin@biospace.com and on LinkedIn.   

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