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FDA Approves First-Ever Drug for a Rare and Aggressive Brain Cancer

Aug 13, 2025

Sriram Venneti, Md, PhDResearch from the labs of Sriram Venneti, MD, PhD, Al and Robert Glick Family Research Professor of Pediatrics, Scientific Research Director of the Chad Carr Pediatric Brain Tumor Center, Department of Pathology and Carl Koschmann, MD, Associate Professor and the ChadTough Defeat DIPG Research Professor, Department of Pediatrics originally published in 2023 has lead the U.S. Food and Drug Administration (FDA) to accelerate approval of the drug ONC201 now called dordaviprone for the treatment of diffuse midline glioma (DMG) with an H3 K27M mutation. A rare, aggressive brain tumor that primarily affects children and young adults. This marks the first FDA-approved therapy for this form of cancer.

Approved on August 6, 2025, dordaviprone is available for adult and pediatric patients aged one year and older whose cancer has progressed despite prior treatment. DMG with the H3 K27M mutation is notoriously difficult to treat, and until now, patients have had no approved drug therapies targeting this specific tumor type.

Why This Matters

Diffuse midline gliomas are often devastating diagnoses with limited to no treatment options available to patients. This FDA approval is a milestone in potentially helping patients who had no targeted therapy available to them before.

This breakthrough may be the first step toward turning a previously untreatable brain cancer into one with real treatment possibilities.

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