Clinical EfficacyPhase 3 SAPPHIRE trial demonstrated statistically significant and clinically meaningful improvements in motor function for spinal muscular atrophy patients, supporting apitegromab's therapeutic benefit independent of SMN-targeted treatments.
Commercial ReadinessReserved capacity at a reputable fill-finish partner combined with active pre-launch outreach to prescribers and the patient community positions the therapy for a coordinated commercial rollout if authorization is achieved.
Regulatory Engagement And PathwayA constructive Type A meeting with FDA indicated the primary approvability issue is facility compliance rather than clinical data, and management plans to resubmit the regulatory application using an alternative fill-finish partner in 2026 to pursue approval and launch.