Experimental drug tested for rare genetic lung disease
Disease control
Terminated
This study tested whether the drug VX-864 could help people with a specific genetic form of Alpha-1 antitrypsin deficiency. Researchers enrolled 14 participants with the PiZZ genotype to receive the drug for 48 weeks. The trial was terminated early, and researchers measured chang…
Phase: PHASE2 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Mar 25, 2026 14:08 UTC