The FDA cleared Scholar Rock’s apitegromab (Isembyld) for spinal muscular atrophy, adding a first-in-class, muscle-targeted option designed to improve motor function in adults and children age 2 and older who are receiving survival motor neuron (SMN)-targeted therapy. The approval follows a prior regulatory delay linked to manufacturing issues, and it reorders the SMA treatment landscape by combining a myostatin pathway approach with existing SMN therapies. Separately, the FDA approved Telix Pharmaceuticals’ Pixclara (floretyrosine F-18, 18F-FET), the first US-approved FET-PET imaging agent for glioma—intended to help differentiate recurrent or progressive tumor from treatment-related changes. Together, the approvals underscore two near-term themes for biotech: regulators are willing to clear new mechanisms in rare disease when evidence is sufficient, and they are also extending clinical imaging tools that can sharpen decision-making in oncology care pathways.