Harpreet Singh: FDA Grants First Systemic Therapy Approval for Rare Pediatric Brain Tumor
Harpreet Singh, Chief Medical Officer at Precision For Medicine, shared on LinkedIn:
“Earlier this month, the FDA granted accelerated approval to dordaviprone (Modeyso, Jazz Pharmaceuticals) for adult and pediatric patients (≥1 year of age), with H3 K27M–mutant diffuse midline glioma (DMG), a devastating and ultra-rare brain tumor that primarily impacts children and young adults. Modeyso was developed by Chimerix, a Jazz Pharmaceuticals Company prior to its acquisition by Jazz Pharmaceuticals in April 2025.
This marks the first FDA-approved systemic therapy for DMG, a milestone for patients and families who have long faced this disease with no FDA-approved systemic treatment options.
The approval was based on patients treated across five open-label clinical trials.
For confirmation, the ACTION trial is ongoing! Patients with newly diagnosed H3 K27M–mutant diffuse glioma who have completed standard frontline radiotherapy are being randomized 1:1:1 to receive placebo, once-weekly dordaviprone, or twice-weekly dordaviprone on two consecutive days. The trial is an international randomized, double-blind, placebo-controlled, parallel-group study.
In this episode of CMO Confidential, I discuss the results and share my perspective on what this approval means for the neuro-oncology community, the expedited approval pathways used for this treatment, and the patients and families who have been waiting for options.”
More articles featuring Harpreet Singh.
-
Challenging the Status Quo in Colorectal Cancer 2024
December 6-8, 2024
-
ESMO 2024 Congress
September 13-17, 2024
-
ASCO Annual Meeting
May 30 - June 4, 2024
-
Yvonne Award 2024
May 31, 2024
-
OncoThon 2024, Online
Feb. 15, 2024
-
Global Summit on War & Cancer 2023, Online
Dec. 14-16, 2023