Vertex's Casgevy Breaks Ground with First Commercial Patient Infusions as Company Readies Another Major Launch for 2025

Vertex Pharmaceuticals has initiated commercial sales of its cystic fibrosis transmembrane conductance regulator (CFTR) modulator, Casgevy (verinurad), following the drug's approval by the US Food and Drug Administration (FDA) in November 2022.

The first commercial patient infusions of Casgevy were administered in January 2023, marking a significant milestone for Vertex and the CF community. Casgevy is the first and only CFTR modulator approved for the treatment of people with CF who have at least one copy of the F508del mutation, the most common CF-causing mutation.

Vertex is also preparing for the potential launch of another CFTR modulator, VX-121, in 2025. VX-121 is currently in Phase 3 clinical trials, and if approved, it will be the first CFTR modulator approved for the treatment of people with CF who have two copies of the F508del mutation.

The commercial launch of Casgevy and the potential launch of VX-121 in 2025 represent major advancements in the treatment of CF. These drugs have the potential to significantly improve the lives of people with CF by reducing the severity of their symptoms and improving their overall health.

The Approval of Casgevy

The FDA approved Casgevy based on data from two Phase 3 clinical trials, which showed that the drug significantly improved lung function and reduced the number of pulmonary exacerbations in people with CF who have at least one copy of the F508del mutation.

In one of the trials, Casgevy was shown to improve lung function by 4.0 percentage points, compared to placebo, at 24 weeks of treatment. In the other trial, Casgevy was shown to reduce the number of pulmonary exacerbations by 30%, compared to placebo, at 52 weeks of treatment.

The Potential Launch of VX-121

VX-121 is a next-generation CFTR modulator that is currently in Phase 3 clinical trials. If approved, VX-121 will be the first CFTR modulator approved for the treatment of people with CF who have two copies of the F508del mutation.

Data from the Phase 3 trials of VX-121 are expected to be released in 2024. If the data are positive, Vertex plans to file for FDA approval of VX-121 in 2025.

The Impact of Casgevy and VX-121

The commercial launch of Casgevy and the potential launch of VX-121 in 2025 represent major advancements in the treatment of CF. These drugs have the potential to significantly improve the lives of people with CF by reducing the severity of their symptoms and improving their overall health.

The approval of Casgevy is a major milestone for Vertex and the CF community. It is the first CFTR modulator approved for the treatment of people with CF who have at least one copy of the F508del mutation, and it has the potential to significantly improve the lives of these patients.

The potential launch of VX-121 in 2025 is another major milestone for Vertex and the CF community. If approved, VX-121 will be the first CFTR modulator approved for the treatment of people with CF who have two copies of the F508del mutation, and it has the potential to significantly improve the lives of these patients.