The U.S. Food and Drug Administration has granted accelerated approval to Jazz Pharmaceuticals’ Modeyso (dordaviprone) for adults and children aged one year and older with recurrent diffuse midline glioma harboring an H3 K27M mutation. The decision makes dordaviprone the first systemic therapy cleared in the United States for this ultra-rare and aggressive brain tumor, which affects an estimated 2,000 patients each year, many of them pediatric. Regulators based the green light on an integrated analysis of 50 patients treated across five open-label studies. The overall response rate was 22% as assessed by blinded independent central review, and responders showed a median duration of response of 10.3 months. Continued approval may hinge on verification of clinical benefit in the ongoing Phase 3 ACTION trial evaluating the drug in newly diagnosed patients following radiotherapy. Modeyso is an oral small-molecule protease activator administered once weekly. Fatigue, headache, vomiting, nausea and musculoskeletal pain were the most common adverse reactions; serious events occurred in 33% of the broader 376-patient safety pool, with hypersensitivity and QT-interval prolongation highlighted in the label. Jazz said commercial supply will be available within weeks and plans an investor briefing on 27 August to detail launch and development plans.