Vertex secures European approval for CRISPR cell therapy

The first CRISPR/Cas9 gene-edited therapy has been granted a marketing authorisation by the European Commission (EC). Vertex Pharmaceuticals’ CASGEVY™ (exagamglogene autotemcel [exa-cel]) is conditionally approved individuals 12 years and over with severe sickle cell disease characterised by recurrent vaso-occlusive crises or transfusion-dependent beta thalassemia.

The cell therapy is indicated in those for whom hematopoietic stem cell (HSC) transplantation is appropriate and a human leukocyte antigen matched related HSC donor is not available.

Significance of the novel approval

This conditional authorisation in the EU makes CASGEVY the only genetic therapy approved for sickle cell disease and transfusion-dependent beta thalassemia in the EU. Over than 8,000 patients are now potentially eligible for the cell therapy treatment.

Crucially, “CASGEVY offers the potential of a functional cure,” declared Dr Franco Locatelli, PhD, Principal investigator in the CLIMB-111 and CLIMB-121 studies, Professor of Pediatrics at the Catholic University of the Sacred Heart, Rome, and Director of the Department of Pediatric Hematology and Oncology at the Bambino Gesù Children’s Hospital.

“This is a historic moment. This approval means patients with these two life-shortening diseases may now be eligible for a one-time treatment. This scientific progress potentially gives people living with these diseases a chance of a future free of the hallmark challenges of their disease,” David Altshuler, Chief Scientific Officer and Executive Vice President, Global Research at Vertex Pharmaceuticals shared with EPR.

The European Commission’s decision is an “important regulatory milestone” principally because of the “significant burden” of these conditions across Europe, Samarth Kulkarni, Chairman and Chief Executive Officer of CRISPR Therapeutics remarked.

About the CRISPR/Cas9 gene-edited cell therapy

CASGEVY™ is a non-viral, ex vivo gene-edited cell therapy for eligible sickle cell disease or transfusion-dependent beta thalassemia patients. The treatment is produced via the editing of a patient’s own hematopoietic stem and progenitor cells.

The change is made at the erythroid specific enhancer region of the BCL11A gene through a precise double-strand break. As a result, high levels of foetal haemoglobin are produced in red blood cells, Vertex Pharmaceuticals confirmed.

Research has shown that CASGEVY has demonstrated an ability to lower or eliminate vaso-occlusive crises in sickle cell disease and transfusion requirements for patients with transfusion-dependent beta thalassemia, according to Vertex Pharmaceuticals.

First CRISPR-based gene-editing therapy authorised