The U.S. Food and Drug Administration (FDA) has approved Modeyso (dordaviprone) for the treatment of patients with diffuse midline glioma with a H3 K27M mutation.
Glial tumors are a type of brain tumor originating in glial cells. Diffuse midline H3 K27M-mutant glioma is a rare, highly aggressive tumor that is more often found in children than adults. The symptoms of glial tumors vary by type but may include headaches, nausea and vomiting, confusion, personality changes, trouble with balance, vision problems, speech difficulties, and/or seizures. The exact underlying cause is unknown.
Dordaviprone is a protease activator of the mitochondrial caseinolytic protease P and inhibitor of dopamine D2 receptor. The compound targets and activates the integrated stress response, induces apoptosis, and alters mitochondrial metabolism, restoring histone H3 K27 trimethylation. It is an orally administered small molecule given once weekly indicated for patients 1 year of age and older with diffuse midline H3 K27M-mutant glioma with progressive disease following prior therapy. It is the first and only treatment approved by the FDA for this indication.
The efficacy of dordaviprone was assessed across five open-label, non-randomized clinical trials in adults and pediatric patients with glioma. The FDA approval follows an integrated efficacy analysis of 50 patients with recurrent H3 K27M-mutant diffuse midline glioma selected from the clinical trials. The overall response rate with dordaviprone was 22% and 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.
The safety of dordaviprone was evaluated in 376 patients with glioma across four open-label clinical trials. Serious adverse events were recorded in 33% patients and included hydrocephalus, vomiting, headache, seizure, and muscular weakness. The most common adverse reactions in patients receiving dordaviprone were fatigue, headache, vomiting, nausea, and musculoskeletal pain.
For more information, click here.
To learn more about gliomas and other rare cancers, visit https://checkrare.com/diseases/cancers/