CAMBRIDGE, Mass. — 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 it has resubmitted the Marketing Authorisation Application (MAA) for apitegromab for the treatment of children and adults with spinal muscular atrophy (SMA) to the European Medicines Agency (EMA).
Informed by ongoing dialogue with the EMA, the resubmitted apitegromab MAA includes Scholar Rock’s alternate fill-finish facility in place of the Catalent Indiana fill-finish facility. The alternate fill-finish facility is the same site that supported the U.S. FDA approval of ISEMBYLD (apitegromab-mstn) in September 2026.
“With the launch of ISEMBYLD now underway in the U.S., we are working with urgency to bring this first-ever muscle-targeted therapy to children and adults living with SMA in Europe,” said David L. Hallal, Chairman and Chief Executive Officer of Scholar Rock. “Consistent with our ongoing dialogue with EMA, we are pleased with the expeditious resubmission of our apitegromab MAA. This marks a decisive step forward in advancing our mission to bring apitegromab to the more than 35,000 SMA patients globally who are receiving an SMN-targeted therapy.”
Apitegromab, marketed as ISEMBYLD™ in the United States, is the first and only muscle-targeted treatment to demonstrate motor function improvement in individuals with SMA currently receiving a survival motor neuron 2 (SMN2)-targeted treatment. In the Phase 3 randomized, placebo-controlled SAPPHIRE study, individuals receiving apitegromab 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.
In August 2026, Scholar Rock informed the Committee for Medicinal Products for Human Use (CHMP) of the withdrawal of its previous MAA due to a lack of GMP compliance at the Catalent Indiana fill-finish facility. With the inclusion of the alternate facility in the MAA resubmission, the Company anticipates a CHMP opinion in the first half of 2027.
Scholar Rock’s alternate fill-finish facility for apitegromab is a U.S.-based facility producing numerous commercial products with a strong track record of regulatory compliance, including recent successful FDA and EMA inspections. Robust supply from this facility is available, with vials on site at a third-party provider awaiting packaging and labeling for commercialization upon approval from the European Commission.
About Apitegromab
Apitegromab is a fully human monoclonal antibody inhibiting myostatin activation by selectively binding the pro- and latent forms of myostatin in the skeletal muscle. It is the first muscle-targeted treatment in spinal muscular atrophy (SMA) to demonstrate clinical success in a pivotal Phase 3 clinical trial. Myostatin, a member of the TGFβ superfamily of growth factors, is expressed primarily by skeletal muscle cells, and the absence of its gene is associated with an increase in muscle mass and strength in multiple animal species, including humans. In the Phase 3 SHAPPHIRE study, apitegromab treatment resulted in clinically meaningful improvements in motor function in patients with SMA on background survival motor neuron 2 (SMN2)-targeted therapy as compared to patients receiving an SMN2-targeted therapy alone In September 2026, the U.S. Food and Drug Administration (FDA) approved apitegromab, under the brand name ISEMBYLD™ (apitegromab-mstn) in the United States, for the treatment of SMA in adults and pediatric patients 2 years of age and older who are currently receiving a SMN2-targeted treatment. Prior to its U.S. approval, apitegromab was granted Fast Track, Orphan Drug and Rare Pediatric Disease designations from the FDA for the treatment of SMA. The European Medicines Agency (EMA) has granted Priority Medicines (PRIME) and Orphan Medicinal Product designations to apitegromab for the treatment of SMA.
About Scholar Rock
Scholar Rock is delivering muscle-targeted breakthroughs to transform the treatment of spinal muscular atrophy (SMA) and other rare neuromuscular diseases where muscle atrophy remains a critical unmet need. Following FDA approval in September 2026, Scholar Rock is commercializing ISEMBLYD (apitegromab-mstn) in the U.S. for people living with SMA who are 2 years of age and older and currently receiving a survival motor neuron 2 (SMN2)-targeted treatment. The company plans to expand access to ISEMBYLD globally, in up to 50 countries worldwide.
As a global leader in myostatin biology, a field focused on proteins that regulate muscle mass, the biopharmaceutical company is named for the visual resemblance of a scholar rock to protein structures. Our commitment to unlock fundamentally different treatment approaches is powered by broad application of a proprietary platform, which has developed novel monoclonal antibodies to modulate protein growth factors with extraordinary selectivity. Scholar Rock works every day to create new possibilities for patients through its highly innovative anti-myostatin program, including opportunities in additional rare neuromuscular diseases. Learn more about our neuromuscular franchise at ScholarRock.com and follow @ScholarRock on X and on LinkedIn.
Scholar Rock® is a registered trademark of Scholar Rock, Inc.
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