Vertex, CRISPR nab world’s first approval for CRISPR-edited therapy

Nov. 16, 2023

The U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted the first regulatory authorization of a CRISPR-based gene-editing therapy in the world to Vertex Pharmaceuticals and CRISPR Therapeutics.

Exa-cel, branded Casgevy, is a CRISPR/Cas9 gene-edited therapy for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). Casgevy was given conditional marketing authorization by MHRA for the treatment of eligible patients 12 years of age and older with SCD with recurrent vaso-occlusive crises or TDT, for whom a human leukocyte antigen matched related hematopoietic stem cell donor is not available. According to Vertex, there are an estimated 2,000 patients eligible for Casgevy in the U.K.

“I hope this represents the first of many applications of this Nobel Prize winning technology to benefit eligible patients with serious diseases,” said Samarth Kulkarni, CEO of CRISPR Therapeutics.

The drug is the first therapeutic product arising from a 2015 collaboration between CRISPR Therapeutics and Vertex. It is currently under review by the European Medicines Agency, the Saudi Food and Drug Authority and the U.S. FDA. 

In the recent FDA AdComm meeting, members closely focused on the data concerning off-target risks, and eventually expressed reservations about Vertex's capacity to evaluate risk across a wide population. However, when it came to efficacy, there were fewer concerns among the members.

The FDA has granted Priority Review for SCD and Standard Review for TDT and assigned PDUFA target action dates of December 8, 2023 and March 30, 2024, respectively.