Resubmission of Apitegromab MAA to EMA for Treatment of Children and Adults with Spinal Muscular Atrophy – Scholar Rock

Scholar Rock 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 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.