FDA Approves Brain Tumor Drug Targeting New Mutation

The FDA granted accelerated approval to dordaviprone (Modeyso) as the first systemic therapy for adults and children with diffuse midline glioma harboring H3 K27M mutations.

Labeling stipulates use in individuals 1 year and older with progressive disease following prior therapy for the rare tumor type, which affects an estimated 2,000 kids and adults in the U.S. annually.

Support for the approval of dordaviprone, a protease activator, was based on results from an integrated efficacy population of 50 adult and pediatric patients with recurrent H3 K27M-mutant diffuse midline glioma enrolled across five open-label, non-randomized trials in the U.S.

Of these, 22% responded per blinded independent central review using Response Assessment in Neuro-Oncology (RANO) 2.0 criteria, with an addit