Overview

• The FDA granted accelerated approval to dordaviprone (Modeyso) for adult and pediatric patients with progressive H3K27M-mutant diffuse midline glioma.
• The disease historically had no approved systemic therapies beyond radiation and palliative measures and carries a median survival of about one year.
• Approval was based on pooled analysis from five single-arm trials showing a 22% overall response rate and a median duration of response of 10.3 months.
• Sixty percent of patients experienced treatment-related side effects—most commonly fatigue, nausea, and lymphopenia—and the prescribing information includes warnings for hypersensitivity, QTc prolongation, and embryo-fetal toxicity.
• Jazz Pharmaceuticals, which acquired dordaviprone through its purchase of Chimerix in early 2025, is recruiting patients for a confirmatory phase 3 trial.