Vertex receives FDA approval for expanded CASGEVY use in children

• The FDA approved expanded use of CASGEVY (exagamglogene autotemcel) for people ages 2 years and older with sickle cell disease or transfusion-dependent beta thalassemia.
• CASGEVY is the first approved genetic therapy for children as young as 2 years for both indications.
• Vertex (NASDAQ:VRTX) established a network of over 75 authorized treatment centers in the U.S. to offer the therapy.

Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) announced that the U.S. Food and Drug Administration has approved expanded use of CASGEVY (exagamglogene autotemcel) for the treatment of people ages 2 years and older with either sickle cell disease with recurrent vaso-occlusive crises or transfusion-dependent beta thalassemia.

CASGEVY is the first approved genetic therapy indicated for children as young as 2 years for both SCD and TDT.

“Just as we redefined what is possible in cystic fibrosis, our ambition is to transform the future for people living with sickle cell disease and transfusion-dependent beta thalassemia,” said Reshma Kewalramani, M.D., Chief Executive Officer and President, Vertex.

The approval expands access to CASGEVY, a one-time CRISPR-based gene-editing therapy.

Vertex has established a network of more than 75 activated authorized treatment centers in the U.S. to offer CASGEVY to eligible patients.

Vertex Pharmaceuticals is a global biotechnology company focused on developing transformative medicines for serious diseases using innovative approaches including gene editing.

The company continues to advance its portfolio of therapies for genetic diseases and other serious conditions.