WebJun 8, 2024 · Vertex Pharmaceuticals announced on Wednesday that the U.S. Food and Drug Administration granted Breakthrough Therapy Designation to its candidate, inaxaplin (VX-147). Inaxaplin was granted the designation for the treatment of APOL1-mediated focal segment glomerulosclerosis (FSGS). WebDec 1, 2024 · – Treatment with VX-147 led to a statistically significant, substantial and clinically meaningful mean reduction in proteinuria of 47.6% at 13 weeks compared to baseline and was well tolerated – – Results provide clinical proof of concept for the first genetically targeted approach to treating kidney disease –

Publication in New England Journal of Medicine of results from …

WebWe report here the characterization of inaxaplin (also known as VX-147), a selective, oral, small-molecule inhibitor of APOL1 channel function, and its effects on proteinuria in a … WebMar 16, 2024 · today announced publication in the New England Journal of Medicine (NEJM) of results from preclinical studies and a Phase 2 study evaluating the efficacy and safety of inaxaplin (VX-147) on top... ctbs480.prw protheus

Vertex Kidney Disease Drug Scores FDA Breakthrough Therapy ... - BioSpace

WebJun 9, 2024 · About the Inaxaplin (VX-147) Pivotal Program A randomized, double-blind, placebo-controlled Phase 2/3 adaptive study is ongoing and will first evaluate two doses of inaxaplin for 12 weeks to select a dose for Phase 3 and subsequently evaluate the efficacy and safety of the single, selected dose in the Phase 3 portion of the study. WebJun 8, 2024 · BOSTON--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) has granted … WebApr 9, 2024 · Official Title: A Phase 2a, Open-label, Single-arm, 2-Part Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of VX-147 in Adults With APOL1-mediated Focal … earshots instructions