Jazz Pharmaceuticals has received the US Food and Drug Administration (FDA) accelerated approval for Modeyso (dordaviprone) to treat diffuse midline glioma with an H3 K27M mutation.
The FDA approval applies to both adult and paediatric patients aged one year and older who have progressive disease following prior therapy.
Modeyso is a small molecule that acts as a protease activator and dopamine receptor inhibitor to induce apoptosis and alter mitochondrial metabolism.
It is the first treatment approved by the FDA for this aggressive brain tumour.
Continued approval is contingent on results from the ongoing Phase 3 ACTION trial, evaluating safety and clinical benefit of Modeyso in newly diagnosed patients.
Chimerix, a Jazz Pharmaceuticals company, chief scientific officer Joshua Allen said: “We’re proud to deliver precisely the kind of transformative innovation we strive for, and we congratulate our combined Chimerix and Jazz team, and the community who worked together tirelessly to bring this treatment forward.
“This approval not only equips clinicians with the first targeted option for this disease but also signals a meaningful shift in what patients and families can expect after diagnosis.
“We would like to extend our thanks to the patients, advocates, clinicians, principal investigators, scientists, regulators and partner institutions who made this possible.”
The FDA’s decision was based on an efficacy analysis of 50 patients with recurrent H3 K27M-mutant diffuse midline glioma.
The overall response rate was 22%, with a median response duration of 10.3 months.
Safety was assessed in 376 patients, with serious adverse reactions occurring in 33% of cases. Common adverse reactions included fatigue, headache, and nausea.
Dana-Farber Cancer Institute center for neuro-oncology director and Harvard Medical School neurology professor Patrick Wen said: “For the first time, we have an FDA-approved therapy for patients with recurrent H3 K27M-mutant diffuse midline glioma.
“While outcomes remain challenging for many patients, the objective responses observed with dordaviprone, including durable benefit in some patients, represent a meaningful advancement.
“This therapy was developed with the underlying biology of the tumour in mind and introduces a new treatment option for a population with historically limited choices.”
Recently, Jazz received the European Commission (EC) conditional marketing authorisation for Ziihera (zanidatamab) to treat a type of biliary tract cancer (BTC). Ziihera is a dual HER2-targeted bispecific antibody that works by binding to extracellular domains 2 and 4 on separate HER2 monomers.
