CHMP issues positive option for first gene-editing medicine

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for the conditional approval of Vertex’s gene-editing therapy Casgevy™ for the treatment of severe sickle cell disease and transfusion-dependent beta thalassemia.

Casgevy (exagamglogene autotemcel) is a CRISPR/Cas9 gene-edited cell therapy. It consists of autologous CD34+ haematopoietic stem cells edited ex vivo by CRISPR/Cas9 technology at the erythroid-specific enhancer region of the BCL11Agene.

When the edited cells are transplanted back into the patients, BCL11A expression is reduced, increasing γ‑globin expression and production of foetal haemoglobin (HbF).

A form of the oxygen-carrying haemoglobin that is naturally present during foetal development, HbF switches to the adult form of haemoglobin after birth. This addresses absent haemoglobin A in patients with TDT and the aberrant haemoglobin S in patients with SCD, according to EMA.

Paving the way for a wave of gene-editing treatments

“Today’s positive CHMP opinion for Casgevy for severe sickle cell disease and transfusion-dependent thalassemia marks a seminal moment in the history of biotechnology and human health,” said Tim Hunt, Chief Executive Officer, Alliance for Regenerative Medicine.

“As the first gene-editing medicine that would be approved in the EU, Casgevy represents a durable and potentially curative treatment option for patients with two serious rare genetic diseases,” Hunt added.

If approved by the European Commission, Casgevy will be eligible for use in patients aged 12 years and older with severe sickle cell disease or transfusion-dependent beta thalassemia, for whom hematopoietic stem cell transplantation is appropriate and a human leukocyte antigen matched related donor is not available.

An approval decision by the European Commission is expected in February 2024.

The milestone will also pave the way for a coming wave of next-generation gene-editing treatments for a range of diseases, from other rare disorders to cancers, Hunt stated.

The positive European opinion, follows a world first approval by the UK Medicines and Healthcare products Regulatory Agency (MHRA) in November. Casgevy was also approved by the US Food and Drug Administration (FDA) for use in patients with sickle cell disease earlier in December. 

Regulatory-first for gene editing therapy