Novartis' del-brax shows promise as first disease-modifying treatment for FSHD after Phase I/II success

Novartis announced that its investigational drug del-brax met primary and key secondary endpoints in the Phase I/II FORTITUDE study for facioscapulohumeral muscular dystrophy (FSHD), showing reductions in biomarkers linked to muscle damage. Del-brax, an antibody oligonucleotide conjugate, targets the root cause of FSHD by suppressing DUX4 gene expression, potentially becoming the first disease-modifying treatment for this rare neuromuscular disease. The company plans to engage global regulators on these results and is currently enrolling patients in a Phase III trial to further evaluate efficacy and safety. This progress marks a significant step toward addressing an unmet medical need affecting tens of thousands in the US and EU.