After FDA meeting, Scholar Rock to again seek apitegromab approval
Agency rejected initial application for SMA drug; developer to resubmit in 2026
After a “constructive and collaborative in-person” meeting with the U.S. Food and Drug Administration (FDA), Scholar Rock is now planning to once again seek approval of its muscle-strengthening agent apitegromab as a treatment for spinal muscular atrophy (SMA).
That resubmission to the FDA is expected in 2026, the developer stated in a company press release following its meeting with the regulatory agency. First, Scholar Rock will work with its third-party fill-finish facility Catalent Indiana — which is tasked with the final steps of the drug’s packaging, such as ensuring the product is sterile — to address concerns raised by the FDA.
Scholar Rock’s announcement comes just a few months after the FDA rejected the company’s initial application for apitegromab, citing manufacturing issues. The agency did not note any problems with the experimental therapy’s safety or efficacy, but said it could not give approval due to issues at the facility. Catalent is now implementing a remediation plan, according to Scholar Rock.
“We are unwavering in our commitment to bring apitegromab, the world’s first and only muscle-targeted treatment to improve motor function, to children and adults living with SMA,” said David L. Hallal, chairman and CEO of Scholar Rock.
Scholar Rock met last week with the FDA, along with representatives from Cure SMA and Catalent (a part of Novo Nordisk). Per the developer, the fill-finish facility expects to be ready for inspection by the end of the year. Scholar Rock would then resubmit its application, with the goal of making apitegromab commercially available to SMA patients before the end of 2026.
“We are grateful to the FDA, Cure SMA, and our colleagues at Novo Nordisk for the positive engagement at our … meeting [last] week,” Hallal said. “We are encouraged by the discussion and by our shared understanding of the urgency to bring this important treatment to the SMA community as rapidly as possible.”
In tandem with its work with Catalent, Scholar Rock has also enlisted a second fill-finish facility to help speed its timelines. The company did not name the second facility, but said that it is “a commercially approved facility that is based in the U.S. and has a proven track record of successful site inspections.”
Apitegromab now under review in EU for approval for SMA
Scholar Rock has also applied for approval of apitegromab for use in the European Union. Review of that application is ongoing, with a decision expected in mid-2026, the company said.
SMA is a genetic disease caused by mutations that result in low levels of SMN, a protein that’s essential for the survival of motor neurons, which are the nerve cells that control movement. Lacking SMN, motor neurons sicken and die, resulting in symptoms like muscle weakness.
There are several approved SMA therapies that boost SMN protein levels; these medicines can help slow or stop disease progression. Nonetheless, many patients still experience persistent muscle weakness that interferes with day-to-day life.
Apitegromab is designed to block the activity of myostatin, a protein that normally helps limit muscle growth. By blocking this protein, the therapy aims to accelerate muscle growth and boost muscle strength.
Scholar Rock’s applications seeking approval of apitegromab were based on positive data from the Phase 3 SAPPHIRE clinical trial (NCT05156320), which tested the therapy against a placebo in more than 180 children and young adults with SMA type 2 or type 3. All were taking the SMN-targeting therapies Spinraza (nusinersen) or Evrysdi (risdiplam). The study met its main goal of showing that apitegromab was significantly better than the placebo at improving a standardized measure of motor function, and no serious side effects were reported.
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