Michael Yeh

Michael Yeh asked about expectations for apitegromab's label, specifically regarding ambulatory/non-ambulatory patients and age subgrouping, and how to think about comparable pricing for a weight-based drug.

Answer

Akshay Vaishnaw, President of R&D, stated that prior advanced label discussions with the FDA suggest a broad label, leveraging data from both non-ambulatory and ambulatory patients, and those two years and older. David Hallal, Chairman and Chief Executive Officer, noted that pricing specifics would be shared post-approval, but key factors include SMA's rarity/severity, progressive nature, and compelling clinical benefits.

Michael Yeh inquired about expectations for apitegromab's label regarding ambulatory/non-ambulatory status and age subgrouping, and how to approach pricing for a weight-based drug compared to other therapies.

Answer

Akshay Vaishnaw, President of R&D, Scholar Rock, anticipated a straightforward alignment on the label given prior progress and the sole CMC issue, noting the package includes ambulatory/non-ambulatory patients two years and older. David Hallal, Chairman and CEO of Scholar Rock, stated pricing specifics are premature but will consider SMA's rarity, severity, progressive nature, and compelling clinical benefits.