Scholar Rock Reports the US FDA's BLA Submission for Apitegromab to Treat Spinal Muscular Atrophy (SMA)
Shots:
- The US FDA received BLA, plus a request for a priority review of apitegromab to improve motor function in pts with SMA who are on SMN-targeted therapy. The EMA’s MAA to be filed in Q1’25
- The BLA was supported by the P-III (SAPPHIRE) & P-II (TOPAZ) trial, where SAPPHIRE met its 1EP of 1.8-point improvement in HFMSE compared to PBO in 52wks. (topline data shared in 2024), with additional findings to be presented at the MDA Clinical & Scientific Conference 2025
- Additionally, apitegromab will be evaluated in the P-II (OPAL) trial for pts (<2yrs.) who are on approved SMN therapy (nusinersen, risdiplam, or onasemnogene abeparvovec), with the study to begin in mid-2025
Ref: Scholar Rock | Image: Scholar Rock
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