Posted by Michael Wonder on 29 Jan 2025
Scholar Rock submits biologics license application to the US FDA for apitegromab as a treatment for patients with spinal muscular atrophy
29 January 2025 - Scholar Rock’s BLA submission is based on the Phase 3 SAPPHIRE trial that demonstrated a statistically significant improvement in motor function for patients receiving apitegromab compared to placebo, as measured by the Hammersmith Functional Motor Scale-Expanded at week 52.
Scholar Rock today announced the submission of a biologics license application to the US FDA for apitegromab, a muscle-targeted therapy designed and developed to provide clinically meaningful improvement in motor function for people living with spinal muscular atrophy who are receiving SMN-targeted treatments.
