FDA Grants Accelerated Approval to Dordaviprone for Patients With Diffuse Midline Glioma
The US Food and Drug Administration (FDA) granted accelerated approval to the protease activator dordaviprone for the treatment of adult and pediatric patients with diffuse midline glioma with an H3 K27M mutation and progressive disease after previous therapy. This regulatory decision was based on data from 5 clinical trials conducted (ONC006, ONC013, ONC014, ONC016, and ONC018).
These open-label, non-randomized trials overall included 50 adult and pediatric patients with recurrent H3 K27M-mutant diffuse midline glioma who were at least 90 days post-radiation therapy and had an adequate washout period from any prior anticancer therapies. Patients included in the efficacy population received dordaviprone monotherapy. The major efficacy outcome measure was overall response rate (ORR) as assessed by blinded independent central review. A secondary outcome measure was duration of response (DOR).
Among the efficacy population, the ORR was 22% and the median DOR was 10.3 months. There were 11 patients with objective responses, 73% of which had a DOR that was ≥ 6 months and 27% of which had a DOR that was ≥ 12 months.
The recommended dosage for dordaviprone for adults is 625 mg orally once weekly. For pediatric patients, the recommended dosage is based on body weight. The prescribing information for dordaviprone includes warnings/precautions for hypersensitivity, QTc interval prolongation, and embryo-fetal toxicity.
Source:
FDA grants accelerated approval to dordaviprone for diffuse midline glioma. Accessed August 6, 2024. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-dordaviprone-diffuse-midline-glioma
