FDA Delays Scholar Rock's SMA Drug Approval Due to Manufacturing Issues at Novo Nordisk Plant

What's Happening?

The U.S. Food and Drug Administration (FDA) has issued a complete response letter (CRL) to Scholar Rock, delaying the approval of its lead drug for spinal muscular atrophy (SMA), apitegromab. The delay is attributed to unresolved compliance issues at a manufacturing plant operated by Novo Nordisk, specifically Catalent Indiana LLC in Bloomington. The FDA's inspection revealed quality issues such as pest infestations, contamination, and equipment failures. Scholar Rock's drug itself was not cited for any safety or efficacy concerns. The company is working closely with Catalent Indiana to address these issues and plans to resubmit the Biologics License Application (BLA) for apitegromab. The drug showed significant improvement in motor function in the SAPPHIRE trial, which tested it in patients aged two to twelve already receiving standard SMA therapies.

Why It's Important?

The delay in FDA approval for apitegromab is significant as it impacts patients with SMA, a genetic disorder that leads to rapid loss of motor neurons affecting muscle functions. Apitegromab is positioned as the first muscle-targeted treatment for SMA, offering potential improvements in motor function beyond existing therapies. The delay also affects Scholar Rock financially, as evidenced by a nearly 13% drop in its Nasdaq share value following the news. The broader implications include potential setbacks in the availability of innovative treatments for SMA patients and financial repercussions for the company and its stakeholders.

What's Next?

Scholar Rock is focused on resolving the manufacturing issues with Catalent Indiana to resubmit the BLA for apitegromab. The company is committed to working with the FDA to secure approval for the drug. Outside the U.S., apitegromab is under review by the European Medicines Agency (EMA), with a decision expected next year. If approved, Scholar Rock plans to launch the drug in Germany as its first European market. The resolution of manufacturing issues and subsequent approval processes will be closely watched by investors and the SMA community.