FDA Commissioner Marty Makary reportedly attempted to intervene to block approval of KalVista Pharmaceuticals' rare disease therapy, raising concerns about agency independence under new leadership. This has added to ongoing scrutiny of FDA regulatory unpredictability, which some analysts argue advantages large pharma over emerging biotech firms. Additionally, landmark reviews of rare disease drug approvals are anticipated to test FDA commitment to biomarker-based accelerated pathways and may impact drug development for pediatric neurological conditions and vaccines.