Summary
Vertex Pharmaceuticals Inc. (VRTX) announced a significant development on December 8, 2023, with the U.S. Food and Drug Administration (FDA) approval of CASGEVY™ (exagamglogene autotemcel). This groundbreaking therapy is an autologous, genome-edited hematopoietic stem cell-based gene therapy designed for the treatment of sickle cell disease (SCD) in patients aged 12 and older who experience recurrent vaso-occlusive crises. This approval marks a major milestone for Vertex and offers a potentially transformative treatment option for a patient population with high unmet medical needs.
Key Highlights
- 1FDA approval of CASGEVY™ (exagamglogene autotemcel) for sickle cell disease (SCD).
- 2CASGEVY is a genome-edited hematopoietic stem cell-based gene therapy.
- 3Indicated for patients aged 12+ with recurrent vaso-occlusive crises.
- 4The wholesale acquisition cost for CASGEVY in the United States has been set at $2.2 million.
- 5This approval represents a significant advancement in genetic therapies for SCD.
- 6Represents a new therapeutic modality for Vertex in its pipeline.