The U.S. Food and Drug Administration (FDA) has expanded the approved use of Casgevy, allowing children as young as 2 years old to receive the gene therapy for the one-time treatment of sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta thalassemia (TDT).
Casgevy, developed by Vertex and co-developed by CRISPR Therapeutics, was previously approved for patients aged 12 years and older. According to Vertex, the expanded indication makes an additional 5,500 children in the United States eligible for treatment.
The label expansion was granted through the FDA’s Commissioner’s National Priority Voucher (CNPV) program. Vertex said the broader approval gives younger patients and their families an opportunity to reduce the risk of organ damage that can result from either disorder.
Support for the decision came in part from findings generated in two ongoing Phase 3 clinical studies. One trial enrolled 11 children between the ages of 5 and 12 who had sickle cell disease. According to a separate FDA approval release, eight participants were evaluable for efficacy, and all eight achieved the primary endpoint of having no severe vaso-occlusive crises for at least 12 consecutive months.
A second trial evaluated Casgevy in 15 children aged 5 to 12 with transfusion-dependent beta thalassemia. Among the nine patients who were evaluable for efficacy, eight achieved the same standard. The median duration of transfusion independence among those patients was reported as 20 months.
Vertex stated that the approval for use in children down to 2 years of age was also supported by the known characteristics of Casgevy and clinical study data in both approved indications.
Haydar Frangoul, Medical Director of HCA Healthcare’s Sarah Cannon Transplant and Cellular Therapy Program at TriStar Centennial Children’s Hospital, commented on the expanded access. He said, “Earlier access to the transformative potential of this therapy will allow clinicians and families to consider treatment before years of cumulative damage from these life-shortening diseases take hold.”
Vertex Chief Executive Officer Reshma Kewalramani said the consistency of results across age groups reinforces the potential of Casgevy to deliver durable benefits to people who have historically had limited options.
The approval was issued under the CNPV program established by former FDA commissioner Marty Makary, who announced his resignation in May during his tenure at the agency. The program was designed to significantly shorten review timelines for certain products and companies aligned with U.S. national interests.
Casgevy first received FDA approval in 2023 for sickle cell disease. At the time, it was described as the first medicine developed using the CRISPR gene-editing system, which earned its inventors a Nobel Prize in 2020 and has potential applications for other diseases that currently lack treatments.
The same day that Casgevy was originally approved, the FDA also approved bluebird bio’s gene therapy Lyfgenia for sickle cell disease. The source information noted that uptake of both therapies has been slow, in part because of their respective prices of $2.2 million and $3.1 million.
Vertex reported Casgevy sales of $116 million in 2025, with 64 patients receiving infusions during the year, including 30 in the fourth quarter. During the first quarter of this year, Casgevy generated $43 million in sales. Vertex also reported that more than 500 patients have initiated treatment since the therapy reached the market.
For Lyfgenia, more than 150 patients completed initial cell collection in 2025 and more than 100 received infusions, according to Genetix Biotherapeutics, which does not report sales of the treatment. After bluebird was sold last year, private equity firms Carlyle and SK Capital established Genetix. Lyfgenia remains approved for patients with SCD and TDT who are 12 years of age and older.
Casgevy has received expanded approval from the U.S. Food and Drug Administration (FDA), allowing its use in eligible children aged 2 years and older with sickle cell disease and transfusion-dependent beta thalassemia. The expanded indication marks an important milestone for Casgevy, increasing access to an innovative gene-editing therapy for younger patients living with serious inherited blood disorders.
Casgevy Receives Expanded FDA Approval
The latest FDA decision broadens the approved patient population for Casgevy, enabling physicians to consider the therapy for younger children who meet the treatment criteria. The expanded approval reflects growing confidence in the clinical evidence supporting Casgevy and its potential to address the underlying genetic causes of these life-threatening diseases.
The decision also represents continued progress in the advancement of gene-editing therapies for rare inherited disorders.
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