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

14 Feb 2024
www.pmlive.com
Gene TherapyDrug Approval
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 EUVertex PharmaceuticalsitingCRISPR Therapeuticshich its inventors were awtransfusion-dependent beta thalassaemia (TDT)sickle cell disease (SCD)
Casgevy bexagamglogene autotemcelh genetic diseases caused by errors in the genes for haemoglobin, used by red blood cells to carry oxygen around the body.
SCD thosbeta-thalassaemiagenetic 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-thalassSCDia, the error can lead to anaemia, with patients who are affected by thevaso-occlusive crises (VOCs)ease requiring regular blood transfusions and iron chelation therapy throughout their lives.pain
Casbeta-thalassaemiato edit the faulty gene anaemiaents’ 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.
Casgevyre now more than 8,000 patients who are potentially eligible for treatment with Casgevy following the regulator’s decision, the companies said.SCDTDT
Reshma Kewalramani, Vertex’s chief executive officer and president, said: “With this apCasgevy 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 firstVertexment to emerge from Vertex and CRISPR Therapeutics' strategic partnerCasgevyriginally announced iSCD015. Vertex now leads the global development, manufacturing and commercialisation of the therapy and splits programme costs and profits worldwide with CRISPR.
Casgevypany said it is already “working closelVertexh natCRISPR Therapeuticsities “to secure access for eligible patients as quicklVertexossible”.
“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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