European review of SMA therapy awaits results from upcoming US facility check
Scholar Rock prepares a secondary manufacturing site for apitegromab
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EU approval for apitegromab, an experimental SMA therapy, awaits US FDA inspection results of a manufacturing facility.
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Apitegromab is an add-on therapy for SMA types 2 and 3, shown to improve motor function.
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Scholar Rock is preparing a second manufacturing facility to ensure supply and address regulatory concerns.
An official decision on whether to approve apitegromab for spinal muscular atrophy (SMA) in the European Union may hinge on upcoming inspection results from U.S. regulators, biopharmaceutical company Scholar Rock has announced.
The European Medicines Agency (EMA) is currently reviewing the experimental muscle-strengthening therapy. However, European authorities are awaiting an official inspection update from the U.S. Food and Drug Administration (FDA) about one of the drug’s key manufacturing facilities before rendering a final opinion.
“We are very pleased with the EMA’s review of the apitegromab [application] and with the level of engagement we have from the Agency,” David L. Hallal, board chair and CEO of Scholar Rock, said in a company press release.
SMA is a neuromuscular disorder that causes progressive muscle weakness. Apitegromab is an antibody-based therapy designed to boost muscle strength by blocking the activity of myostatin, a protein that normally prevents excess muscle growth. Scholar Rock is developing apitegromab as an add-on to disease-modifying treatments that slow progression by targeting SMA’s root cause, with the aim of helping patients achieve better motor function.
Phase 3 trial and regulatory path
A Phase 3 clinical trial called SAPPHIRE (NCT05156320) tested apitegromab against a placebo in children and young adults with SMA type 2 or type 3 who were taking the disease-modifying therapies Spinraza (nusinersen) or Evrysdi (risdiplam). The study met its main goal, demonstrating that motor function scores improved significantly in patients receiving apitegromab.
Based on those positive Phase 3 results, Scholar Rock submitted an application seeking U.S. approval for apitegromab in early 2025. However, later that year, the FDA declined to approve the initial submission. The agency raised no concerns about apitegromab’s safety or efficacy data, but it cited compliance issues at a third-party fill-finish facility, a specialized manufacturing center responsible for final drug packaging and product sterility.
Since receiving the FDA’s decision, Scholar Rock has been working with the facility, known as Catalent Indiana (part of Novo Nordisk), to address regulators’ concerns. To ensure a stable medication supply, Scholar Rock also enlisted a second fill-finish facility. Scholar Rock has since resubmitted its U.S. application, and an FDA decision is expected in late September.
Meanwhile, Scholar Rock is also seeking approval of apitegromab in the EU. Notably, while the company’s resubmitted U.S. application details both fill-finish facilities, the European application currently lists only the Catalent Indiana site.
When experimental therapies are reviewed in the EU, a key step is evaluation by the Committee for Medicinal Products for Human Use (CHMP), an arm of the EMA. The committee makes recommendations for or against approval, which are almost always followed by the European Commission, the body with final decision-making authority for EU drug approvals.
Scholar Rock prepares dual-facility backup
The FDA completed an inspection of the Catalent Indiana facility in April, and according to Scholar Rock, the CHMP is withholding its recommendation on apitegromab until the FDA’s inspection results are available.
If the FDA gives the facility the green light, an opinion from the CHMP is expected before the end of 2026. If not, Scholar Rock said it will work with European regulators to update its European filing to incorporate the second fill-finish facility, which the company said is already in good standing with both the FDA and EMA.
“We expect clarity from the FDA on the inspection classification of Catalent Indiana in the near term, and we remain confident in our ability to advance our [application for apitegromab] through the EMA review process either with Catalent Indiana or by taking the necessary steps to include our second fill-finish facility,” Hallal said.
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