CAMBRIDGE (MASSACHUSETTS) (dpa-AFX) - The European Commission has granted
conditional marketing authorization to Casgevy, a CRISPR/Cas9 gene-edited
therapy. Casgevy is approved for the treatment of patients who are 12 years of
age and older with severe sickle cell disease characterized by recurrent
vaso-occlusive crises or transfusion-dependent beta thalassemia, for whom
hematopoietic stem cell transplantation is appropriate and a human leukocyte
antigen matched related hematopoietic stem cell donor is not available, Vertex
Pharmaceuticals Inc. (VRTX) said in a statement on Tuesday.

The company noted that more than 8,000 patients 12 years of age and older with
severe sickle cell disease or transfusion-dependent beta thalassemia may be
eligible for treatment.

Vertex said it has secured early access for eligible transfusion-dependent beta
thalassemia patients in France ahead of the national reimbursement process. The
company continues to engage with hospitals experienced in stem cell
transplantation to establish a network of independently operated authorized
treatment centers (ATCs) for the administration of CASGEVY. There are currently
three activated ATCs in the EU and Vertex plans to activate a total of
approximately 25 centers across Europe.

In December 2023, Vertex said that the European Medicines Agency's (EMA)
Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion
for the conditional approval of Casgevy (exagamglogene autotemcel (exa-cel)), a
CRISPR/Cas9 gene-edited therapy, for the treatment of severe sickle cell disease
and transfusion-dependent beta thalassemia.

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