EC approves Vertex/CRISPR’s Casgevy gene therapy for inherited blood disorders

14 Feb 2024
www.pmlive.com
Gene TherapyDrug Approval
EC approves Vertex/CRISPR’s Casgevy gene therapy for inherited blood disorders
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Source: PMLiVE
The European Commission (EC) has granted conditional marketing authorisation to Vertex Pharmaceuticals and CRISPR Therapeutics’ gene therapy for use in transfusion-dependent beta thalassaemia (TDT) and severe sickle cell disease (SCD) patients aged 12 years and over.
Casgevy (exagamglogene autotemcel) is now the first treatment approved in the EU that uses the gene-editing tool CRISPR, for which its inventors were awarded the Nobel Prize in 2020.
SCD and beta-thalassaemia are both genetic diseases caused by errors in the genes for haemoglobin, used by red blood cells to carry oxygen around the body.
For those with SCD, this genetic error can lead to organ damage, a shortened lifespan and vaso-occlusive crises (VOCs), which are caused by blockages of blood vessels by sickled red blood cells and result in severe and debilitating pain.
In beta-thalassaemia, the error can lead to anaemia, with patients who are affected by the most severe form of the disease requiring regular blood transfusions and iron chelation therapy throughout their lives.
Casgevy is designed to edit the faulty gene in patients’ bone marrow stem cells so that the body produces functioning haemoglobin. The therapy has been specifically authorised by the EC for use in SCD patients with recurrent VOCs or those with TDT for whom haematopoietic stem cell transplantation is appropriate but not available.
There are now more than 8,000 patients who are potentially eligible for treatment with Casgevy following the regulator’s decision, the companies said.
Reshma Kewalramani, Vertex’s chief executive officer and president, said: “With this approval, Casgevy is now approved for SCD and TBT in multiple geographies, making tens of thousands of patients eligible for this potentially transformative therapy.”
Casgevy is the first treatment to emerge from Vertex and CRISPR Therapeutics' strategic partnership, originally announced in 2015. Vertex now leads the global development, manufacturing and commercialisation of the therapy and splits programme costs and profits worldwide with CRISPR.
The company said it is already “working closely” with national health authorities “to secure access for eligible patients as quickly as possible”.
“Now our goal shifts to translating these approvals into real-world patient [benefits] and ensuring access and reimbursement across the globe,” Kewalramani said.
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