The FDA has granted accelerated approval to dordaviprone for the treatment of diffuse midline glioma with an H3 K27M mutation in patients one year old and older with progressive disease following therapy.
This is the first FDA-approved systemic therapy for this kind of brain tumor, which is known for being aggressive and difficult to treat, according to an Aug. 6 news release from the agency.
The approval was based on data from 50 patients enrolled across five open-labeled, nonrandomized clinical trials conducted in the U.S. All patients had measurable disease, had received prior radiation and other anticancer therapies and were assessed using Response Assessment in Neuro-Oncology (RANO) criteria for high-grade glioma.
The overall response rate was 22% with a median response of 10.3 months. Out of the 11 patients who responded, 73% maintained a response for at least six months and 27% for at least 12 months.
