Novartis’ $12bn Avidity Buyout Validated by FSHD Biomarker Trial Win
NVS•Novartis’ $12bn acquisition of Avidity Biosciences is justified as delbacibart braxlosiran achieved its primary endpoint in a 90-patient Phase I/II FSHD trial, showing significant drops in creatine kinase and KDHC1L biomarkers. These results validate the dosing regimen for the ongoing 200-patient Phase III FORTITUDE-3 study, with data due Q3 2028.
1. Mid-Stage FSHD Trial Results
In the Phase I/II FORTITUDE study involving 90 patients with facioscapulohumeral muscular dystrophy, delbacibart braxlosiran met its primary endpoint by significantly reducing key disease biomarkers creatine kinase and KDHC1L. This strong target engagement replicates earlier dose-escalation findings and underscores the therapy’s potential to reduce muscle damage in FSHD.
2. Validation of Phase III Design and Timeline
The positive biomarker outcomes validate the dosing regimen for the multinational Phase III FORTITUDE-3 trial, which aims to enroll 200 patients. Novartis plans to report top-line data from this pivotal study in the third quarter of 2028, positioning delbacibart braxlosiran for regulatory review upon successful completion.
3. Pipeline Impact and Market Potential
If late-stage results confirm efficacy and safety, delbacibart braxlosiran could become the first approved therapy for FSHD, a rare disease affecting 45,000–87,000 patients in the US and EU. This milestone would strengthen Novartis’ neuromuscular portfolio and outpace competing programs such as Scholar Rock’s apitegromab in FSHD trials.
