Scholar Rock (SRRK) Jefferies Global Healthcare Conference 2026 summary

Regulatory and commercial progress

• Apitegromab is under review by the FDA with a PDUFA date of September 30 and by the EMA, with a CHMP decision expected around mid-year.

• Plans are in place to expand regulatory submissions to up to 50 countries beyond the U.S. and Europe.

• The opportunity is seen as multi-billion dollar annually, with little meaningful competition expected for 5–10 years.

• Launch preparations in the U.S. and Europe are complete, with teams ready for immediate rollout upon approval.

• European launch will begin in Germany, with other countries following over several years due to reimbursement processes.

Manufacturing and regulatory strategy

• Two independent fill-finish facilities (Catalent Indiana and a second U.S.-based site) are included in the BLA, providing dual paths to approval.

• Catalent Indiana underwent FDA re-inspection in April 2026, resulting in a Form 483 with eight observations; Novo Nordisk responded within 15 business days.

• FDA decision on Catalent’s status is expected by late July, determining if the facility will be reclassified and can be used for approval.

• The second fill-finish facility was rapidly qualified, with all launch drug supply already vialed and ready for release upon approval.

• FDA alignment was achieved through multiple meetings, ensuring both facilities meet regulatory requirements and leveraging stability data from Catalent.

Inspection outcomes and risk mitigation

• Persistent issues at Catalent Indiana include particulate matter and mammalian hair, though improvements have reduced contamination by 85%.

• Novo Nordisk’s remediation plan included halting and then resuming operations to focus on compliance.

• FDA is reviewing Novo’s responses; no further inspection is anticipated before a decision.

• The second facility’s process and materials closely match Catalent’s, enabling rapid tech transfer and regulatory acceptance.

• Release testing is performed at a third-party site, expediting qualification of the new facility.