FDA rejects Scholar Rocks SMA drug, citing issues at Catalent Indiana site
8.4
来源：
FiercePharma
发布时间：
2025-09-23 09:22
摘要：
Scholar Rock's apitegromab, aimed at treating spinal muscular atrophy (SMA), faced an FDA rejection due to manufacturing issues at a third-party facility. Despite this setback, the drug has shown promise in improving motor function in SMA patients, particularly when combined with Eli Lilly's tirzepatide. The company plans to address the FDA's concerns and resubmit its application, with expectations of significant market potential, including projected peak sales of $1.8 billion.
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关键证据
FDA rejection due to manufacturing issues at a third-party facility.
Phase 2 study results show apitegromab improves motor function in SMA patients.
Projected peak sales for apitegromab are estimated at $1.8 billion.
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AI评分总结
Scholar Rock's apitegromab, aimed at treating spinal muscular atrophy (SMA), faced an FDA rejection due to manufacturing issues at a third-party facility. Despite this setback, the drug has shown promise in improving motor function in SMA patients, particularly when combined with Eli Lilly's tirzepatide. The company plans to address the FDA's concerns and resubmit its application, with expectations of significant market potential, including projected peak sales of $1.8 billion.