2024-02-07 15:05:33 ET
Summary
- Vanza CF triplet achieved non-inferiority compared to TRIKAFTA in lung function measure and achieved statistically significant improvement in sweat chloride levels compared to it.
- Regulatory applications of Vanza triplet to the FDA and EMA to treat CF patients ages 6 and older with the F508del mutation or responsive to CFTR modulator therapy, expected mid-2024.
- A Priority Review Voucher will be used for Vanza with respect to the FDA filing, so that it could obtain a shorter review period for possible U.S. marketing approval.
- Data from a phase 1 multiple-ascending dose study, using nebulized mRNA VX-522 for the treatment of patient with 5,000 CF patients that wouldn't benefit from CFTR modulator therapy, expected late 2024 or early 2025.
Vertex Pharmaceuticals ( VRTX ) continues to make significant progress in being able to advance treatment options for patients with Cystic Fibrosis [CF]. It just reported positive results from several late-stage studies, using its next-generation CF triplet vanzacaftor/tezacaftor/deutivacaftor for the treatment of CF patients. It was shown that this triplet was non-inferior to TRIKAFTA in treating CF patients who have at least one F508del mutation or responsive to triple combination CFTR modulators [CFTRm]. Such non-inferiority of the Vanza triplet was observed in the two phase 3 studies, known as SKYLINE-102 and SKYLINE-103. One area of CF that Vanza did achieve superiority on, which moves the bar forward for these patients, was being able to show sweat chloride level improvements for them....
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Vertex: Vanza Triplet Data Allows For Mid-2024 Regulatory Submissions