Scholar Rock Submits FDA Application for Apitegromab to Improve Motor Function in Spinal Muscular Atrophy

Scholar Rock Submits FDA Application for Apitegromab to Improve Motor Function in Spinal Muscular Atrophy

Apitegromab is a muscle-targeted therapy for improvement of motor function in individuals with spinal muscular atrophy.

Scholar Rock submits application of SMA therapy to FDA

Additionally, the company is on track to submit a marketing authorisation application (MAA) to the European Medicines Agency (EMA) in the first quarter of this year. The Division The post Scholar Rock submits application of SMA therapy to FDA appeared first on Pharmaceutical Business review.

BLA Submitted for Muscle-Promoting Therapy Apitegromab as New Treatment for Spinal Muscular Atrophy

The submission was based on promising data from a phase 3 and phase 2 study, where treatment with apitegromab led to significant improvements in motor function among patients with SMA on SMN-targeted treatments.

Scholar Rock Submits Biologics License Application (BLA) to the U.S. FDA for Apitegromab as a Treatment for Patients with Spinal Muscular Atrophy (SMA) - Pipelinereview

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 The FDA has granted apitegromab Fast Track, Orphan Drug and Rare Pediatric Disease Designations in SMA Scholar Rock remains on track to submit the apitegromab Marketing Autho…

Scholar Rock Submits Biologics License Application (BLA) to the U.S. FDA for Apitegromab as a Treatment for Patients with Spinal Muscular Atrophy (SMA)

“With the strength of our Phase 3 data as the foundation of our submission, we look forward to continuing to work closely with the FDA through the review of our BLA on behalf of patients and families living with SMA.” Today, Scholar Rock announced the submission of a Biologics License Application (BLA)  to the U.S. Food and Drug Administration (FDA) for apitegromab, a muscle-targeted therapy designed and developed to prov…