Scholar Rock (SRRK) Status update summary

Regulatory update and BLA resubmission

• Resubmitted the Biologics License Application (BLA) for Apitegromab to the FDA, targeting children and adults with spinal muscular atrophy (SMA), including both Catalent Indiana and a second U.S.-based fill-finish facility, following FDA guidance from the March 2026 Type C meeting.

• FDA alignment allowed resubmission prior to Catalent Indiana's reinspection, with no additional corrective actions requested after the recent site visit, reflecting significant remediation progress.

• Anticipates FDA acceptance of the BLA within 30 days and a review period of up to six months, with a potential PDUFA action date in late September 2026.

• The BLA update was limited in scope, mainly a safety update and draft labeling from prior FDA interactions.

• European Medicines Agency (EMA) review of the Marketing Authorization Application is progressing, with a decision anticipated mid-2026.

Manufacturing and supply chain flexibility

• Both fill-finish facilities are included in the BLA based on their individual progress and FDA guidance, strengthening supply chain and supporting global commercial plans.

• If one facility is removed from the BLA, a supplemental BLA (sBLA) will be submitted for the other to ensure supply chain robustness.

• Commercial Apitegromab from the second facility will be available several months before the anticipated PDUFA date.

• The second facility has a strong inspection history and is expected to meet all regulatory requirements within the review window.

• Inventory planning ensures sufficient supply for launch, regardless of which facility is approved first.

Commercial and launch readiness

• Launch preparations are advanced, with a focus on seamless patient support and robust supply chain.

• Specialty pharmacy and home infusion networks have been expanded to support broad patient access.

• Engagement with payers and healthcare providers is ongoing to ensure broad coverage and efficient patient transition.

• Pricing strategy considers disease rarity, severity, and Apitegromab’s clinical benefit, with minimal impact expected from global pricing reforms.

• The team is fully staffed and financially prepared for launch, with no material increase in cash burn anticipated before approval.