Jazz Pharmaceuticals' Modeyso Approved by NCCN for Rare Brain Cancer Treatment

Jazz Pharmaceuticals announced that its cancer treatment, Modeyso, has been added to the National Comprehensive Cancer Network guidelines for a rare form of brain cancer. Jazz Pharmaceuticals is a biotechnology company that develops medicines for serious diseases, including Xywav, Xyrem, Epidiolex, Rylaze, Zepzelca, Defitelio, and Vyxeos.

Jazz Pharmaceuticals plc (Nasdaq: JAZZ) announced that its cancer treatment, Modeyso™ (dordaviprone), has been included in the National Comprehensive Cancer Network® (NCCN®) Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for H3 K27M-mutant diffuse glioma. Modeyso is now recommended as a category 2A single-agent treatment option for pediatric and adult patients with recurrent or progressive diffuse high-grade glioma harboring an H3 K27M mutation .

Modeyso was granted accelerated approval by the U.S. Food and Drug Administration (FDA) on August 6, 2025, 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 . The FDA approval was based on an integrated efficacy analysis of 50 patients from five open-label clinical studies, showing an overall response rate (ORR) of 22% and a median duration of response of 10.3 months .

The NCCN Guidelines® play a pivotal role in cancer care decision-making, providing expert recommendations for screening, diagnosis, and treatment. The inclusion of Modeyso in the guidelines reflects the urgency of the unmet need for effective treatments in H3 K27M-mutant diffuse midline glioma, a rare and aggressive brain tumor with a poor prognosis .

Modeyso is an orally administered small molecule that activates the integrated stress response, induces apoptosis, and alters mitochondrial metabolism, leading to restored histone H3 K27 trimethylation in H3 K27M-mutant diffuse glioma . The drug received accelerated approval based on pre-specified efficacy data and is currently undergoing further evaluation in the Phase 3 ACTION trial (NCT05580562) .

Jazz Pharmaceuticals acquired the rights to Modeyso from Chimerix in April 2025. Modeyso is not approved anywhere else in the world . The company's portfolio includes other cancer treatments such as Xywav, Xyrem, Epidiolex, Rylaze, Zepzelca, Defitelio, and Vyxeos.

References:
https://www.morningstar.com/news/pr-newswire/20250909sf68074/modeyso-dordaviprone-included-in-national-comprehensive-cancer-network-nccn-clinical-practice-guidelines-in-oncology-for-h3-k27m-mutant-diffuse-glioma