FDA Approvals in 2024: Novel Therapies for MASH, WHIM Syndrome, Pediatric Glioma, MDS, and Bladder Cancer
• The FDA approved Madrigal's Rezdiffra, the first treatment for metabolic dysfunction-associated steatohepatitis (MASH), addressing a significant unmet need in liver disease.
• X4 Pharmaceuticals' Xolremdi gained approval as the first targeted therapy for WHIM syndrome, a rare immunodeficiency, marking a milestone for the company.
• Day One Biopharmaceuticals' Ojemda secured approval for BRAF-altered pediatric low-grade glioma, offering a new treatment option for this common childhood brain tumor.
• Geron Corporation's Rytelo, a telomerase inhibitor, received FDA approval for lower- to intermediate-risk myelodysplastic syndromes (MDS), after 34 years in business.
• ImmunityBio's Anktiva, a novel IL-15 superagonist, was approved for non-muscle invasive bladder cancer, providing a new therapeutic approach for BCG-unresponsive patients.
© Copyright 2025. All Rights Reserved by MedPath