VRTX · 10-Q · 2026Q3 · Full report

CASGEVY Pediatric Market Expansion

VERTEX PHARMACEUTICALS INC / MA · 2026-11-03 · Importance 72 · Surprise 100

CASGEVY generated $99.2 million in third-quarter 2026 product revenue, up 30% from the second quarter and more than 400% year over year. The FDA approved CASGEVY for children aged two and older with sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT), and multiple children had initiated treatment following approval. The United Arab Emirates and Bahrain approved use in children aged two and older, while the United Kingdom approved treatment for children aged five and older. Vertex completed regulatory submissions in Saudi Arabia for pediatric SCD and TDT.

Key facts

• The FDA approved CASGEVY for children two years of age and older with SCD or TDT, making it the first genetic therapy indicated for children as young as two for those conditions; multiple children have initiated treatment since approval. source
• In October, the United Arab Emirates and Kingdom of Bahrain approved CASGEVY for children aged 2 and older, and the United Kingdom approved it for children aged 5 and older; Vertex completed regulatory submissions in Saudi Arabia for children with SCD or TDT. source