Successful BLA Submission for Apitegromab
Scholar Rock submitted the Biologics License Application (BLA) for apitegromab in the U.S. in January and is on track to submit the Marketing Authorization Application (MAA) in the EU in March.
Positive Results for Apitegromab in SMA
Apitegromab plus standard-of-care delivered a clinically meaningful and statistically significant improvement of 1.8 points on the Hammersmith Functional Motor scale compared to placebo. 30% of patients achieved an additional 3-point or greater improvement.
Commercial Preparation for Apitegromab Launch
Scholar Rock is preparing for a potential Q4 2025 U.S. launch of apitegromab, with plans to scale the customer-facing team and engage with U.S. commercial and federal payers.
Strong Engagement with SMA Community
Scholar Rock has introduced the 'Life Takes Muscle' campaign to raise awareness and has been engaging with leading CureSMA centers and neurologists.