Novartis' $12B acquisition shows promise as del-brax meets key trial endpoints for rare muscle disease FSHD

Novartis announced that its investigational therapy del-brax met primary and key secondary endpoints in a Phase 1/2 trial for facioscapulohumeral muscular dystrophy (FSHD), a rare neuromuscular disease. The therapy showed reductions in biomarkers linked to muscle damage, indicating effective target engagement and muscle protection. Del-brax, acquired through Novartis' $12 billion purchase of Avidity Biosciences, is now in Phase 3 trials and holds orphan drug designations from the FDA and EMA. This progress supports Novartis' strategy to develop treatments for rare neuromuscular disorders and plans to engage regulators as development continues.