Casgevy approval unlikely to be followed up by another CRISPR drug in near future

However, given that 88% of CRISPR drugs are in the early stages of development, it is unlikely to see another CRISPR approval any time soon, says data and analytics company GlobalData.

In November 2023, Casgevy secured the first approval for a CRISPR-based drug from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) for the treatment of sickle cell disease and beta thalassemia.

This decision made history by approving the first genome editing drug. The approval was swiftly followed by the FDA’s approval for sickle cell disease on December 8.

This new treatment offers a potential cure for inherited blood disorders, which are caused by errors in the hemoglobin genes.

Previously, the only permanent treatment option for the blood conditions was a bone marrow transplant, which carries a plethora of risks, including rejection.

Casgevy was developed following a 2015 partnership between Vertex Pharmaceuticals and CRISPR Therapeutics. It works by precisely editing the faulty gene in a patient’s bone marrow stem cell, enabling the production of functional hemoglobin.

Currently, Casgevy is the only marketed CRISPR drug, as well as the only CRISPR drug featuring in pre-registration and phase 3 stages of development.

It is in pre-registration for sickle cell disease in the EU, as well as beta thalassemia in the EU and US. Additionally, Casgevy is in phase 3 for these indications in Canada.

Jasper Morley, drugs intelligence analyst at GlobalData, comments: “Despite a gradual increase in CRISPR drugs from phase 2 compared to phase 1, the earlier stages of development, discovery, and preclinical account for 88% of active CRISPR pipeline drugs. This composition indicates that CRISPR drugs are still very much in their infancy and are yet to be consistently established throughout the later stages of drug development.”

Nevertheless, there are currently 24 CRISPR drugs in phase 2, including CRISPR Therapeutics’s CTX-110.

If successful, GlobalData predicts this drug to be the next CRISPR therapy to be globally launched, towards the end of 2025.

This drug is in phase 2 for oncological indications, such as B-cell acute lymphocytic leukemia. However, according to GlobalData’s likelihood of approval tool, it is predicted that this drug only has a 48% chance of advancing into phase 3 trials, with only an overall 31% likelihood of approval.

Morley concludes: “The Casgevy approvals represent a significant milestone for revolutionary genome editing systems. However, given the relative immaturity of the CRISPR drugs pipeline, which features very few late-stage products alongside a low likelihood of approvals, it is unlikely to see another drug approval in the near future. It will be interesting to see how the success of Casgevy impacts CRISPR research and drug development as time progresses.”