The World’s First CRISPR Gene Therapy Approved in UK!

Witten by Johann Sanséau, Analyst Email:[email protected]

Vertex and CRISPR Therapeutics’ Casgevy receives conditional approval for sickle cell disease and thalassemia in the UK. 

On Thursday morning, the UK became the first country to conditionally approve Vertex and CRISPR Therapeutics’ gene therapy, Casgevy, for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT)1.  

Casgevy (exagamglogene autotemcel or exa-cel) utilizes CRISPR technology to enable patients to produce functional hemoglobin. The therapy has undergone two global clinical trials for SCD and TDT, successfully meeting the primary endpoint in both. Patients treated with Casgevy were free from severe vaso-occlusive crises (VOCs) or achieved transfusion independence for at least 12 months1, with the hope that these effects will be lifelong. 

Casgevy was granted an Innovation Passport (IP) under the Innovative Licensing and Access Pathway (ILAP) by the Medicines and Healthcare products Regulatory Agency (MHRA) in August. The ILAP aims to accelerate the time to market and facilitate patient access to medicines. Over 100 IPs have been awarded since the program’s inception in 20212. As part of the IP, a product-specific team of experts will help to define the target development profile (TDP) of the therapy and create a road map for delivering early patient access. Several therapies, such as Lumykras (sotorasib) and Lorviqua (lorlatinib), received an IP and went on to receive positive recommendations from NICE3,4.  

Potentially due to Casgevy’s IP, Vertex have stated that they are already actively collaborating with national health authorities to expedite access for eligible patients in the UK. According to Vertex, approximately 2,000 patients in the UK are eligible for Casgevy1

Casgevy is being launched in two indications simultaneously, SCD and TDT, which will have its own challenges. Although, as these indications are both haemoglobinopathies, these patients are likely to be in the same treatment centers, simplifying patient access somewhat.  

While the UK has taken the lead in approving Casgevy, the therapy is awaiting a decision from the US Food and Drug Administration (FDA), expected in early December1. Additionally, the European Medicines Agency (EMA) is currently reviewing the gene therapy1. This places Casgevy in a pivotal position as the second gene therapy indicated for TDT attempting commercialization in Europe, following bluebird bio’s Zynteglo. Zynteglo received EMA approval in 2019, however the marketing authorization was later withdrawn by bluebird bio after they failed to reach an agreement with several European payers for its reimbursement5.  

It is important to realize that regulatory approval is only a small step towards commercial success for Casgevy. Zynteglo secured marketing authorization in addition to positive HTAs from several markets, and still withdrew from Europe due to failing to secure an acceptable price in key markets. Once Casgevy undergoes HTAs in the UK and Europe (subject to EMA approval), Vertex’s strategies to secure reimbursement will be key in determining the commercial success of the therapy.

In conclusion, the conditional approval of Casgevy in the UK represents a major breakthrough in the field of gene therapy, being the first CRISPR therapy approved in the world. Casgevy offers an alternative treatment for patients who would otherwise require lifelong transfusions every few weeks. The coming months will be crucial in shaping the future of Casgevy and its impact on patients’ lives.  

P4A will continue to monitor the commercialization efforts of Casgevy to provide up to date and relevant advice to our clients. Reach out to us to learn more about our services and to remain up to date on the opportunities in an ever-changing space.  



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