Scholar Rock shifts manufacturing plan to keep SMA drug on track
Company expects FDA decision on apitegromab by end of September
Written by |
- Scholar Rock will use a second plant for its experimental SMA treatment apitegromab after the FDA found continued problems at a facility.
- Apitegromab is a muscle-strengthening therapy designed to improve motor function.
- The FDA expects to make a decision on the drug by the end of September.
The U.S. Food and Drug Administration (FDA) has once again found problems at a manufacturing facility that was included on Scholar Rock‘s application seeking approval of apitegromab, its experimental muscle-strengthening agent for spinal muscular atrophy (SMA).
In light of the continued issues at the facility, Scholar Rock said it will remove that facility from its application and will proceed with a second plant instead. FDA inspection of the second facility is already in progress, and the company still expects the FDA to issue a decision by the end of September.
“As we work closely with the FDA on the final steps of the apitegromab [application] review process, we are ready to bring the world’s first muscle-targeted treatment to children and adults with SMA,” David L. Hallal, board chair and CEO of Scholar Rock, said in a company press release.
SMA is a genetic disease that causes muscle weakness. Apitegromab is designed to boost muscle strength by blocking the activity of myostatin, a protein that normally prevents excess muscle growth. Scholar Rock is developing it to improve motor function in people with SMA who are taking disease-modifying treatments that target SMA’s underlying cause to slow disease progression.
In early 2025, Scholar Rock submitted an application seeking FDA approval of apitegromab. The application was based on positive data from SAPPHIRE (NCT05156320), a Phase 3 study that tested apitegromab against a placebo in young people with SMA who were taking disease-modifying treatments. Results showed apitegromab outperformed the placebo at improving motor function scores.
Problems at plant
The FDA rejected Scholar Rock’s initial application. The agency didn’t raise any concerns about the clinical data for apitegromab, but issues were found at a fill-finish facility — a manufacturing center that performs the final steps of medication packaging, such as ensuring the product is sterile. Scholar Rock has since been working with the facility, known as Catalent Indiana (part of Novo Nordisk), to address the FDA’s concerns. At the same time, Scholar Rock enlisted a second fill-finish facility in alignment with FDA guidance.
When Scholar Rock resubmitted its application to the FDA, the new application included both Catalent Indiana and the second facility. The FDA conducted a new inspection of Catalent Indiana earlier this year, and Scholar Rock announced that the agency classified the site as Official Action Indicated, meaning there are significant problems that haven’t been adequately addressed.
The FDA advised Scholar Rock to remove Catalent Indiana from the application and proceed with only the second fill-finish facility, and Scholar Rock is planning to do just that. Scholar Rock didn’t identify the second facility but said it is in good standing with the FDA and the European Medicines Agency (EMA). The company has submitted data to the FDA needed for the second facility’s inspection, and the agency is reviewing the facility.
Scholar Rock said the second facility has already produced a robust supply of apitegromab, prepped for labeling and packaging so that the treatment can hit markets as soon as possible if the FDA gives the all-clear.
“We continue to be very pleased with our second fill-finish facility,” Hallal said. “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.”
Scholar Rock has also submitted an application seeking approval of apitegromab in the European Union. However, that application only included the Catalent Indiana fill-finish facility, and regulators in the EU were waiting on the FDA’s inspection of that facility before proceeding with the application. Scholar Rock said it will work with EU regulators to switch its application to the second fill-finish facility.
Leave a comment
Fill in the required fields to post. Your email address will not be published.