VX-983

VX-983 is an investigational small molecule CFTR (cystic fibrosis transmembrane conductance regulator) modulator being developed by Vertex Pharmaceuticals for the treatment of cystic fibrosis (CF). It is designed to target the underlying protein defect in CF, where mutations in the CFTR gene lead to absent or dysfunctional CFTR protein, impairing the transport of salt and water across cell membranes. VX-983 has been evaluated in Phase 1 clinical development, including randomized, double-blind, placebo-controlled dose-escalation studies in healthy adults. These early trials also investigated the drug's pharmacokinetic profile and potential drug-drug interactions, specifically assessing its effect on the metabolism of midazolam, a known CYP3A4 substrate.