The FDA approved Scholar Rock’s Isembyld (apitegromab-mstn), a myostatin inhibitor for spinal muscular atrophy (SMA) in adults and children 2 years and older who are receiving SMN2-targeted therapy. The approval follows a manufacturing-related delay tied to the company’s “fill-finish” provider. The authorization also makes Isembyld the first new commercial SMA option in years to directly target muscle tissue, adding to the existing landscape of gene-targeting and SMN2-modulating therapies. Scholar Rock had previously seen its stock jump in late 2024 after earlier findings that showed meaningful motor-function improvements when added to standard care. Separately, the FDA approved Telix Pharmaceuticals’ Pixclara (floretyrosine F-18, 18F-FET), the first FDA-approved FET-PET imaging agent for glioma, after a complete response letter requested additional clinical evidence. Together, the two approvals show a dual regulatory push: faster access to therapies for hard-to-treat rare disease populations and expanded imaging tools to support earlier differentiation of tumor recurrence versus treatment effects in brain cancer.