The U.S. FDA has granted accelerated approval for Jazz Pharmaceuticals’ oral capsules, Modeyso, for the treatment of adult and pediatric patients 1 year of age and older with diffuse midline glioma harboring an H3 K27M mutation with progressive disease following prior therapy.
Modeyso is the first treatment option for this ultra-rare and aggressive brain tumor, which primarily affects children and young adults. To address the urgent unmet patient need, Modeyso is expected to be commercially available in the coming weeks.
Modeyso is administered as an oral capsule once weekly. The FDA's decision was based on an integrated efficacy analysis of 50 patients with recurrent H3 K27M-mutant diffuse midline glioma, selected from five open-label clinical studies based on prespecified eligibility criteria. The overall response rate, as assessed by blinded independent central review using Response Assessment in Neuro-Oncology (RANO) 2.0 criteria, was 22%. Among responders, the median duration of response was 10.3 months.
Continued approval for the indication is contingent on verification of clinical benefit in the phase 3 ACTION confirmatory trial.