FDA Rejects Scholar Rock's SMA Drug Due to Manufacturing Issues at Novo-Owned Facility

Scholar Rock's bid to introduce the first muscle-targeted treatment for spinal muscular atrophy (SMA) has encountered a significant setback. The U.S. Food and Drug Administration (FDA) has issued a complete response letter (CRL) rejecting the company's application for apitegromab, citing manufacturing concerns at a third-party facility in Indiana.

Manufacturing Hurdles Delay Approval

The FDA's decision stems from observations made during a general site inspection of the Indiana facility, which Novo Nordisk acquired from Catalent as part of a larger $16.5 billion deal finalized in December 2023. Importantly, the regulatory body did not raise any concerns about the efficacy or safety of apitegromab itself.

Scholar Rock emphasized that the issues identified were not specific to apitegromab's production. The company had previously warned investors of potential FDA rejection during its second-quarter earnings presentation, citing problems at two third-party contract manufacturers.

In response to the FDA's concerns, the Indiana facility, referred to as Catalent Indiana, submitted a response in early August and has continued to implement corrective actions. Scholar Rock plans to resubmit its Biologics License Application (BLA) once these issues are resolved, with expectations of an expedited review process.

Implications for Scholar Rock and the SMA Community

The delay affects Scholar Rock's aspirations to launch its first marketed product. As of June 30, the 13-year-old company reported a cash balance of $295 million, with its financial runway extending into 2027. Jefferies analyst Amy Li projects peak sales for apitegromab to reach $1.8 billion.

Apitegromab, an infused monoclonal antibody treatment, aims to address muscle deterioration in SMA patients. It has shown promise in improving motor function when combined with existing treatments that prevent motor neuron loss but do not directly target muscle health.

Kenneth Hobby, President of Cure SMA, underscored the urgency for new treatments, stating, "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."

Broader Industry Impact

This setback for Scholar Rock highlights ongoing challenges in pharmaceutical manufacturing and regulatory compliance. The Indiana facility, now owned by Novo Nordisk, has been linked to other recent regulatory issues, including delays in FDA decisions for Regeneron's high-dose version of Eylea and the rejection of its blood cancer bispecific odronextamab.

As the pharmaceutical industry continues to navigate complex manufacturing and regulatory landscapes, the importance of robust quality control measures and proactive communication with regulatory bodies remains paramount. The outcome of Scholar Rock's resubmission will be closely watched by industry stakeholders and the SMA community alike.