NEW YORK – Chimerix on Tuesday said the US Food and Drug Administration accepted its new drug application for accelerated approval of its investigational cancer therapy dordaviprone as a treatment for patients with recurrent H3 K27M-mutant diffuse glioma and granted it priority review.
Dordaviprone is a member of a class of cancer therapies dubbed imipridones that target G protein-coupled receptors and mitochondrial caseinolytic protease P in cancer cells. In initial findings from Phase III ACTION trial of dordaviprone, following radiotherapy in patients 2 years and older with newly diagnosed H3 K27M-mutant diffuse glioma who have completed standard frontline radiotherapy, the disease control rate was 40 percent and the objective response rate was 28 percent, according to Response Assessment in Neuro-Oncology (RANO) 2.0 criteria. Out of 50 evaluable patients, 10 had a partial response, six had stable disease, and 15 had progressive disease. The median duration of response was 10.4 months. Dordaviprone, formerly known as ONC201, was well tolerated and the majority of treatment-related adverse events were mild.
"Patients with this form of high-grade glioma face a very difficult prognosis with few treatment options beyond palliative care," Chimerix CEO Mike Andriole said in a statement. "Our team is working expeditiously with the FDA to facilitate their review as we simultaneously prepare for a potential commercial launch in order to ensure rapid availability to patients in need."
The FDA has granted rare pediatric disease designation to dordaviprone for H3 K27M-mutant glioma, and the Durham, North Carolina-based firm has applied for a rare pediatric disease priority review voucher in the NDA submission.
The FDA expects to issue a decision on dordaviprone by Aug. 18.