- Trial Name
- RESILIENT
- Age:
- 4 to 21 years old
- Aim:
- To evaluate the efficacy and safety of taldefgrobep alfa in participants with spinal muscular atrophy
- Status:
- Enrollment completed
(14th September 23) - Location:
- Belgium, France, Germany, Italy, Netherlands, Poland, Spain, UK & USA
- Identifier:
- NCT05337553
Clinical Trials
Overview
Taldefgrobep alfa – Biohaven
Taldefgrobep alfa is a muscle-directed therapy, developed by Biohaven that aims to increase muscle mass and strength in people living with SMA.
Method of Action
Taldefgrobep alfa is an anti-myostatin, which means it targets myostatin, a natural protein that is important in healthy muscular development. In people with neuromuscular diseases, active myostatin can limit the skeletal muscle growth needed to achieve developmental and functional milestones. Taldefgropep alfa is designed to increase muscle mass and strength in SMA patients via dual inhibition functioning, by directly lowering myostatin levels and blocking myostatin function.
Trials of taldefgrobep alfa
Community updates
Regulatory:
- Community Update: RESILIENT SMA trial did not meet primary endpoint - December 2024
- Biohaven Completes Enrollment in Pivotal Phase 3 Study of Taldefgrobep Alfa in Spinal Muscular Atrophy- October 2023
- taldefgrobep alfa receives FDA ffast-track designation for spinal muscular atrophy patients in addition to previously receiving orphan drug designation in the US.- February 2023