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Scholar Rock Receives CRL from FDA for Spinal Muscular Atrophy Treatment Apitegromab
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Key Takeaways
- Scholar Rock's Apitegromab BLA received a CRL due to issues at Catalent Indiana's facility, unrelated to the drug itself.
- Scholar Rock is working with Catalent to address FDA observations and plans to resubmit the BLA promptly.
Scholar Rock navigates FDA's Complete Response Letter for Apitegromab, aiming for swift resubmission to address spinal muscular atrophy treatment needs.
The CRL resulted from observations identified at the Catalent Indiana LLC Fill-Finish Facility.
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Scholar Rock announced it has received a Complete Response Letter (CRL) from FDA for Apitegromab’s Biologics License Application (BLA) for the treatment of patients with spinal muscular atrophy (SMA). According to Scholar Rock, the CRL was issued due to observations collected at the Catalent Indiana LLC Fill-Finish Facility during a routine site inspection with the observations listed in the CRL not directed at Apitegromab, along with no additional approvability concerns for the treatment. The fill-finish facility is third-party and was acquired by Novo Nordisk in December 2024.
“We are continuing to work closely with Catalent Indiana on the FDA’s manufacturing observations so that we can resubmit the Apitegromab BLA as soon as possible,” said David L. Hallal, chairman and chief executive officer of Scholar Rock. “We remain focused on working hand-in-hand with the FDA to pursue approval of the first and only muscle-targeted treatment for people living with SMA.”
Scholar Rock’s next steps following the Catalent Indiana fill-finish facility observations
Scholar Rock discussed the Catalent facility observations back in its second quarter 2025 earnings announcement, which was followed by Scholar Rock submitting a comprehensive response earlier in August of this year addressing the observations recorded from FDA. Since Scholar Rock’s submission of the response, the Catalent Indiana fill-finish facility shifted its focus to taking corrective action to improving the observations in the CRL along with keeping FDA informed on its progress.
Following the Catalent Indiana facility’s rectification of FDA’s observations, Scholar Rock will resubmit the Apitegromab BLA, as it anticipates FDA to act swiftly on the application process upon resolving its manufacturing concerns.
What is Apitegromab?
Apitegromab is an investigational fully human monoclonal antibody inhibiting myostatin activated by selectively binding the pro- and latent forms of myostatin in the skeletal muscle.1 Apitegromab is the first muscle-targeted treatment candidate in SMA to demonstrate clinical success in a Phase III clinical trial. Myostatin, a member of the TGFβ superfamily of growth factors expressed primarily by skeletal muscle cells and the absence of its gene is associated with an increase in muscle mass and strength in multiple animal species, including humans.1 Scholar Rock anticipates the highly selective targeting of pro- and latent forms of myostatin paired with Apitegromab could lead to clinical improvements in motor function for patients diagnosed with SMA. FDA granted Apitegromab Fast Track, Orphan Drug and Rare Pediatric Disease designations, along with the European Medicines Agency (EMA) granting it Priority Medicines (PRIME) and Orphan Medicinal Product designations. As of September 2025, Apitegromab has yet to receive approval for any use by the FDA or any other regulatory agency.
“While we are disappointed that the availability of a muscle-targeted treatment approach for patients with SMA has been delayed, we remain enthusiastic about the transformative potential of Apitegromab,” said Kenneth Hobby, president of Cure SMA. “Muscle strength and motor function are significant unmet needs for many in the SMA community and are fundamental to independence. A gain in motor function can allow someone to participate in important activities of daily living from self-care to work and social interactions, and as such, we urgently await the availability of the first-ever treatment with the potential to address the muscular component of SMA.”
Sources
- FDA Issues Complete Response Letter (CRL) for Apitegromab as a Treatment for Patients with Spinal Muscular Atrophy (SMA) Solely Related to Observations Identified at Catalent Indiana LLC Fill-Finish Facility Scholar Rock September 23, 2025 https://investors.scholarrock.com/news-releases/news-release-details/fda-issues-complete-response-letter-crl-apitegromab-treatment
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