• Today, August 7, 2026, Scholar Rock received customer notification from Catalent Indiana LLC (part of Novo Nordisk) that FDA classified the April 2026 site inspection of Catalent Indiana fill-finish facility as Official Action Indicated (OAI)
  • Scholar Rock continuing to collaborate closely with FDA and under their guidance, will remove Catalent Indiana fill-finish facility from apitegromab BLA
  • FDA review of second fill-finish facility progressing; Data package for FDA review of second fill-finish facility, as agreed to during March 2026 Type C meeting, has been submitted in advance of agreed upon timeline
  • Robust supply of apitegromab commercial vials from second fill-finish facility available upon FDA approval
  • Scholar Rock is prepared for U.S. apitegromab launch immediately upon FDA approval, which may be granted at any time through September 30, 2026 Prescription Drug User Fee Act (PDUFA) date
  • Company engaging with European Medicines Agency (EMA) on next steps to include Scholar Rock’s second fill-finish facility in apitegromab Marketing Authorisation Application (MAA)

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 world-leading platform in myostatin biology, today announced that the FDA review of the apitegromab Biologics License Application (BLA) for the treatment of children and adults with spinal muscular atrophy (SMA) is progressing with the Company’s second fill-finish facility. The BLA remains on track for potential FDA approval by the September 30, 2026 Prescription Drug User Free Act (PDUFA) action date.

"We continue to be very pleased with our second fill-finish facility," said David L. Hallal, Board Chair and Chief Executive Officer of Scholar Rock. "The data package that we agreed upon with the FDA at our Type C meeting has been submitted, the FDA review of the facility is advancing, robust supply for commercialization is ready and awaiting packaging and labeling at a third-party site, and importantly, the facility has demonstrated a strong track record of compliance, including recent successful FDA and EMA inspections. As we work closely with the FDA on the final steps of the apitegromab BLA review process, we are ready to bring the world’s first muscle-targeted treatment to children and adults with SMA."

Scholar Rock’s apitegromab BLA submission in March 2026 included two fill-finish facilities, in alignment with FDA guidance. These facilities were Catalent Indiana LLC (part of Novo Nordisk) and a second facility, which provided two independent paths to a potential FDA approval. The Company received a customer notification from Catalent Indiana on August 7, 2026 that the FDA has classified the April 2026 site inspection of Catalent Indiana as Official Action Indicated (OAI). Scholar Rock will continue to collaborate closely with the FDA and under their guidance, will remove Catalent Indiana from the apitegromab BLA. FDA review of the apitegromab BLA will progress solely with the second fill-finish facility.

At the March 2026 Type C meeting, FDA and Scholar Rock agreed to the data package required for FDA review of the second fill-finish facility. That data package was submitted in advance of the agreed upon timeline, and Agency review of the data is progressing well. Robust supply from the second fill-finish facility is available, with vials from that facility on site at a third-party provider awaiting packaging and labeling for commercialization upon FDA approval.

The second fill-finish facility for apitegromab is a U.S.-based facility producing numerous commercial products and it is in good standing with FDA and EMA.

In Europe, Scholar Rock continues to engage with the European Medicines Agency (EMA) on next steps to include the Company’s second fill-finish facility in the apitegromab Marketing Authorisation Application (MAA). The Company plans to provide updated timelines for a Committee for Medicinal Products for Human Use (CHMP) opinion upon alignment with the EMA.