newsfilter
← Latest News
Business Wire·

Scholar Rock Resubmits Apitegromab Marketing Authorisation Application (MAA) to European Medicines Agency (EMA) for Treatment of Children and Adults with Spinal Muscular Atrophy (SMA)

SRRK
  • Scholar Rock resubmitted the Marketing Authorisation Application (MAA) for apitegromab to the European Medicines Agency (EMA) to treat spinal muscular atrophy (SMA) in children & adults.
  • The resubmission replaces the Catalent Indiana LLC fill-finish facility with an alternate U.S. facility aligned with EMA guidance due to prior GMP compliance issues.
  • The alternate fill-finish site supported the U.S. FDA approval of ISEMBYLD (apitegromab-mstn) in September 2026.
  • Scholar Rock anticipates receiving a Committee for Medicinal Products for Human Use (CHMP) opinion in the first half of 2027.
  • Apitegromab is the first muscle-targeted treatment to demonstrate motor function improvement in SMA patients receiving SMN2-targeted therapy in the Phase 3 SAPPHIRE study.
  • Vials from the alternate facility are currently on site at a third-party provider awaiting packaging & labeling for European commercialization upon approval.
  • The European Medicines Agency has granted Priority Medicines (PRIME) & Orphan Medicinal Product designations for apitegromab.
  • ISEMBYLD launched commercially in the U.S. in late 2026 for patients aged 2 years & older who are currently on SMN2-targeted treatment.