FDA Approves Jazz Pharmaceuticals Modeyso to Treat Rare Brain Tumor  

Jazz Pharmaceuticals received accelerated approval from the U.S. FDA for Modeyso (dordaviprone) for the treatment of adult and pediatric patients 1 year and older with diffuse midline glioma harboring an H3 K27M mutation with progressive disease following prior therapy. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the Phase 3 ACTION confirmatory trial.

Modeyso is the first and only treatment option approved by the FDA for this ultra-rare and aggressive brain tumor that affects an estimated 2,000 people in the U.S. each year, many of whom are children and young adults. The disease is characterized by rapid progression and historically has had no effective systemic treatment options. 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. The overall response rate (ORR) was 22%. Among responders, the median duration of response was 10.3 months, with 73% maintaining their response for at least six months and 27% for at least 12 months.