Shots:
- The US FDA has approved Isembyld for the treatment of spinal muscular atrophy (SMA) in pts (≥2yrs.) who are currently receiving a SMN2-targeted treatment
- Approval was backed by the P-III (SAPPHIRE), which met its 1EP of 2.2-point improvement in HFMSE with Isembyld + SMN2-targeted therapy vs PBO at 1yr. in main efficacy population (n=103); 34.2% pts showed a ≥3-point increase in HFMSE vs 13.5%
- Isembyld’s US commercial launch is underway, with product shipments expected in the coming days; Scholar Rock also received a Rare Pediatric Disease Priority Review Voucher for a future marketing application
Ref: Businesswire | Image: Scholar Rock | Press Release
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