Vertex presented the first pediatric clinical data for its gene‑edited therapy CASGEVY (exagamglogene autotemcel) at the American Society of Hematology Annual Meeting. The data, released on December 6 2025, show that all four patients with sickle cell disease (SCD) aged 5‑11 years remained free from vaso‑occlusive crises for at least 12 consecutive months, while all six patients with transfusion‑dependent beta‑thalassemia (TDT) achieved transfusion independence for the same period.
The study, CLIMB‑151 for SCD and CLIMB‑141 for TDT, is the first time a gene‑therapy product has demonstrated safety and efficacy in this younger cohort. The results confirm that the CRISPR/Cas9‑based editing of patients’ own stem cells can sustain high fetal hemoglobin levels, translating into durable clinical benefit in children who previously had limited treatment options.
Vertex’s announcement expands CASGEVY’s approved age range to 5‑11 years, a critical step toward global regulatory submissions slated for the first half of 2026. The company also received a Commissioner’s National Priority Voucher from the U.S. FDA, accelerating review for this pediatric indication. By opening a larger patient population—estimated at several thousand worldwide—Vertex can increase its market reach and reinforce the high‑price, one‑time‑treatment model that underpins the drug’s commercial strategy.
This data milestone supports Vertex’s broader diversification strategy beyond its cystic‑fibrosis core. CASGEVY, priced at $2.2 million per treatment, is a flagship asset in Vertex’s rare‑blood‑disorder portfolio, complementing other pipeline candidates such as JOURNAVX and kidney‑disease programs. The company’s strong cash position and robust pipeline give it the financial flexibility to pursue these growth opportunities while maintaining its leadership in cystic fibrosis.
Dr. Carmen Bozic, Executive Vice President, Global Medicines Development and Medical Affairs, said the results “demonstrate the transformative potential of CASGEVY in children ages 5‑11 years with SCD and TDT.” She added that the pediatric data “will accelerate global regulatory filings and bring the cure closer to patients who need it most.”
Vertex has already secured approvals for CASGEVY in the United States, United Kingdom, European Union, Saudi Arabia, Bahrain, Kuwait, Qatar, Canada, Switzerland, and the United Arab Emirates. The gene‑editing mechanism, which permanently edits the HBB gene to increase fetal hemoglobin, offers a durable cure that can reduce lifetime health‑care costs for patients and payers alike.
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