Scholar Rock (SRRK) Leerink Global Healthcare Conference 2026 summary

Regulatory and manufacturing updates

• Positive phase III SAPPHIRE trial results for Apitegromab in SMA led to a BLA submission in January 2025 and priority review with an action date of September 22, 2025.

• Approval was delayed due to compliance issues at the fill-finish facility in Indiana, now owned by Novo Nordisk, resulting in a complete response letter from the FDA.

• A Type A meeting with the FDA in November 2025 established a sense of urgency and led to a rapid sequence of remediation steps, including a warning letter and a positive site visit.

• Formal re-inspection of the facility is expected soon, with BLA resubmission and U.S. launch anticipated in 2026.

• A second fill-finish facility is being qualified as a contingency, with engineering and validation runs underway to ensure supply chain redundancy.

Supply chain and product readiness

• Tens of thousands of vials are available, supporting ongoing trials and early access programs, with launch inventory ready.

• The Indiana facility continues to manufacture and release approved drugs, and the second facility will provide additional supply in 2026.

• No need for additional manufacturing before launch; both facilities will support the 2026 launch timeline.

Commercial launch and market preparation

• Leadership team with extensive launch experience has been assembled, and commercial planning accelerated after the initial delay.

• Field teams have been hired, trained, and deployed, focusing on disease awareness and education at 140 SMA centers.

• A home infusion network and patient assistance program have been established to support access and reimbursement.

• Ongoing payer engagement and internal training aim to ensure readiness and maintain team sharpness ahead of approval.

• Anticipated payer headwinds at launch, but strong demand expected from patients, families, and physicians.