The FDA has just granted accelerated approval to dordaviprone (Modeyso) for adult and pediatric patients (aged 1 year or older) with diffuse midline glioma harboring an H3 K27M mutation and progressive disease after prior therapy. This marks a milestone as the first systemic therapy approved by the FDA for H3 K27M-mutant diffuse midline glioma.
Who Is Eligible for Dordaviprone?
Patients whose tumors test positive for the H3 K27M mutation and who have diffuse midline glioma that has progressed following earlier treatment may now have access to this new systemic therapy.
Clinical Trial Outcomes
- In clinical studies across 5 trials (50 patients), dordaviprone was tested in adults and children with recurrent H3 K27M-mutant diffuse midline glioma.
- Overall Response Rate (ORR): 22% (95% CI: 12, 36)
- Median Duration of Response (DOR): 10.3 months (95% CI: 7.3, 15.2)
- Of those responding to therapy:
- 73% experienced a response lasting ≥6 months
- 27% had responses lasting ≥12 months
Safety Information
Prescribing guidance for dordaviprone includes warnings about:
- Hypersensitivity reactions
- QTc interval prolongation (potential heart rhythm irregularities)
- Embryo-fetal toxicity (risk for pregnant patients)
Why This Matters: For patients and families facing diffuse midline glioma with H3 K27M mutation—especially after progression despite prior treatment—this FDA approval offers new hope and the potential for meaningful benefit. Ask your oncologist about whether testing for the H3 K27M mutation and dordaviprone therapy are options for your care.
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