Vertex Presents New Data on CASGEVY®, Including First European Presentation of Data in Children Ages 5–11, at the European Hematology Association Congress and Announces Additional Global Regulatory Submissions
Vertex presented Phase 3 data for CASGEVY in children ages 5–11 with severe sickle cell disease or transfusion-dependent beta thalassemia at the European Hematology Association Congress. In the SCD study, all 11 dosed patients were free from vaso-occlusive crises, with 100% of those with sufficient follow-up achieving the primary endpoint of being VOC-free for at least 12 months. In the TDT study, 15 patients were dosed, and 100% of those with sufficient follow-up achieved transfusion independence for at least 12 months while maintaining stable hemoglobin levels.
Key figures
- Drug Name
- CASGEVY (exagamglogene autotemcel)
- Phase Of Trial
- Phase 3
- Scd voc free rate
- 100%
- Scd patients dosed
- 11
- Tdt patients dosed
- 15
- Tdt transfusion independent rate
- 100%
- Scd voc free duration mean months
- 19
- Tdt transfusion independent duration mean months
- 23.4
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