Recently, Biohaven released a statement on the completed enrollment of their pivotal Phase 3 Study of Taldefgrobep Alfa in people with SMA.
Check out Biohaven's official press release here.
About Taldefgrobep and the Phase 3 Trial
Taldefgrobep is an investigational, muscle-targeted recombinant protein with the potential to enhance muscle mass and strength in people living with SMA when used in combination with other approved treatments. Taldefgrobep targets myostatin, a natural protein that limits skeletal muscle growth. Myostatin inhibition is a potential therapeutic strategy for children and adults with a range of neuromuscular conditions for whom active myostatin can limit the skeletal muscle growth needed to achieve developmental and functional milestones.
RESILIENT is a Phase 3 placebo-controlled, double-blind trial designed to evaluate the efficacy and safety of taldefgrobep at 48 weeks as an adjunctive therapy for participants who are already taking a stable dose of nusinersen, risdiplam and/or have a history of treatment with onasemnogene abeparvovec-xioi, compared to placebo. The study is not restricted nor limited to patients based on ambulatory status or classification of SMA.
Taldefgrobep was granted EU Orphan Drug Designation, along with both Fast Track and Orphan Drug Designation by the US FDA.