Vertex Says Casgevy Label Expansion Opens Treatment to 5,500 More Us Children

On Thursday, the U.S.

Food and Drug Administration (FDA) approved an expanded label for Vertex Pharmaceuticals Incorporated ‘s (NASDAQ: VRTX ) Casgevy (exagamglogene autotemcel).

The approval allows Casgevy to be used in patients aged 2 years and older with sickle cell disease (SCD), accompanied by recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta thalassemia (TDT).

The decision makes Casgevy the first approved genetic therapy available for children as young as 2 with either condition.

Read Also: Vertex Says Pediatric CASGEVY Data Show Consistent Benefits In Rare Blood Disorders FDA Decision Broadens Patient Eligibility For Vertex Gene Therapy The expanded indication significantly increases the number of eligible patients in the U.S.

Vertex said approximately 5,500 additional children can now receive the one-time genetic therapy, building on the drug’s previous approval for patients aged 12 years and older.

The latest FDA decision extends access to younger children living with severe sickle cell disease or transfusion-dependent beta thalassemia, two inherited blood disorders that can require lifelong management.

Treatment Network Already In Place Vertex said it has established a nationwide network of independently operated authorized treatment centers to provide CASGEVY to eligible patients through existing access and reimbursement pathways.

According to the company, more than 75 authorized treatment centers across the U.S. are currently activated to administer the therapy, supporting broader patient access following the expanded approval.

International Reviews Continue Beyond the U.S., Vertex said regulatory authorities in the Kingdom of Saudi Arabia and the United Kingdom are currently reviewing applications to expand Casgevy’s label to include younger patients.

The expanded FDA approval marks another milestone for Casgevy, extending the availability of the gene therapy to a broader pediatric population while the company continues to pursue similar regulatory approvals in other markets.

In June, the FDA accepted Vertex’s Biologics License Application submission for povetacicept in adults with immunoglobulin A nephropathy.

The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of November 30, 2026.

If approved, povetacicept will become the first commercialized therapy in Vertex’s emerging nephrology franchise.

VRTX Price Action: Vertex Pharmaceuticals shares were up 0.60% at $501.00 during premarket trading on Thursday.

The stock is trading near its 52-week high of $507.92, according to Pro data.

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