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.
Frequently Asked Questions
CASGEVY is approved for the treatment of sickle cell disease (SCD) in individuals aged 12 years and older who experience recurrent vaso-occlusive crises.
The wholesale acquisition cost for CASGEVY in the United States has been established at $2.2 million.
CASGEVY is an autologous, genome-edited hematopoietic stem cell-based gene therapy.
This FDA approval is highly significant as it marks the first approval of a gene-editing therapy for sickle cell disease, representing a major scientific and commercial advancement for Vertex Pharmaceuticals and offering a new treatment option for patients with this debilitating condition.