Scholar Rock Announces FDA Approval of ISEMBYLD (apitegromab-mstn), the First and Only Muscle-Targeted Treatment for Children and Adults with Spinal Muscular Atrophy (SMA)

Scholar Rock (NASDAQ: SRRK), a global biopharmaceutical company dedicated to improving the lives of patients with rare, severe, and debilitating neuromuscular diseases by applying its leading platform in myostatin biology, today announced that the FDA has approved ISEMBYLD (apitegromab-mstn) for the treatment of spinal muscular atrophy (SMA) in adults and children two years of age and older who are currently receiving a survival motor neuron 2 (SMN2)-targeted treatment.

SMA is a rare, severe neuromuscular disease that results in irreversible loss of motor neurons and progressive muscle wasting, causing continuous motor function decline throughout life and diminishing the independence of both adults and children.

ISEMBYLD is the first and only muscle-targeted treatment to demonstrate motor function improvement in individuals with SMA currently receiving an SMN2-targeted treatment. In the Phase 3 randomized, placebo-controlled SAPPHIRE study, individuals receiving ISEMBYLD demonstrated a robust, clinically meaningful improvement in motor function after one year of treatment while those on an SMN2-targeted treatment alone experienced a loss of motor function.

“Today’s FDA approval of ISEMBYLD marks a defining moment for the SMA community as we now launch the world’s first-ever muscle targeted treatment for children and adults living with SMA in the U.S.,” said David L. Hallal, Chairman and Chief Executive Officer of Scholar Rock. “After decades of failed industry-wide efforts to unlock the potential of myostatin inhibition, Scholar Rock has delivered a therapeutic breakthrough with ISEMBYLD. Our U.S. commercial team is now engaging physicians, SMA care teams, and payers on behalf of the SMA community and our Scholar Rock Supports team stands ready to provide dedicated, comprehensive assistance to patients and caregivers. I would like to extend my deepest thanks to our clinical study investigators, Cure SMA, and other patient advocacy groups for their dedication and support on this journey. Above all, I want to express my heartfelt gratitude to the patients and families affected by SMA who participated in our clinical trials for their trust in Scholar Rock and unwavering resilience every step of the way.”

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