August 07, 2025
1 min read
Key takeaways:
- The FDA approved Modeyso for patients with H3 K27M-mutant diffuse midline glioma.
- The therapy showed an overall response rate of 22%.
The FDA granted accelerated approval to dordaviprone for the treatment of patients aged 1 year and older with recurrent diffuse midline glioma who harbor the H3 K27M mutation with progressive disease following prior treatment.
With this approval, dordaviprone (Modeyso, Jazz Pharmaceuticals) is the only treatment option available for a rare and aggressive brain tumor affecting nearly 2,000 patients in the United States each year, according to the press release.
The FDA issued approval for dordaviprone in patients aged 1 year and older with diffuse midline glioma harboring an H3 K27M mutation.
“This is a major turning point in neuro-oncology,” Patrick Wen, MD, director of the Center for Neuro-Oncology at Dana-Farber Cancer Institute and professor of neurology at Harvard Medical School, said in the release. “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.”
The FDA based its approval of oral once-weekly dordaviprone on an integrated efficacy analysis of 50 patients with recurrent H3 K27M-mutant diffuse midline glioma included in five open-label clinical studies. Results showed an overall response rate of 22% (95% CI, 12-36) in those with progressive disease after prior treatment.
According to the release, median duration of response was 10.3 months (95% CI, 7.3-15.2), with 73% of responders maintaining their response for at least 6 months and 27% for at least 1 year.
“This approval represents a long-awaited treatment option for families affected by H3 K27M-mutant diffuse midline glioma,” David F. Arons, president and chief executive officer of the National Brain Tumor Society, said in the release. “This is a fast-moving, devastating disease that turns families’ lives upside down. For years, this diagnosis has lacked an approved treatment and [now], that changes. Families finally have a treatment option, and a reason to believe in more time together to make memories that might not have otherwise been possible.”
