The Food and Drug Administration (FDA) has fast tracked an investigational therapy for diffuse intrinsic pontine glioma, a rare pediatric brainstem cancer.
Oblato Inc plans to evaluate OKN-007 (disodium 4-[(tert-butyl-imino) methyl] benzene-1,3-disulfonate N-oxide) for diffuse intrinsic pontine glioma in a phase 1/2 trial this year.
The FDA’s Fast Track designation allows for accelerated approval and priority review of drugs that are intended to treat serious conditions and that demonstrate the potential to address an unmet need. Oblato is also eligible for a rare pediatric disease priority review of OKN-007 as the drug has been granted a Rare Pediatric Disease designation.
“We anticipate that the Fast Track designation for DIPG will shorten the NDA review process. The Company will make every effort to develop this treatment for this rare pediatric disease,” stated an official from the Company.
The Company is also investigating OKN-007 combined with temozolomide in patients with recurrent glioblastoma multiforme (ClinicalTrials.gov: NCT04388475). Results from a phase 1b study showed that the novel anticancer agent appears to be safe and may prolong overall survival in patients with recurrent glioma.
- Oblato announces Fast Track designation of OKN-007 for diffuse intrinsic pontine glioma from the FDA. [press release]. Princeton, NJ: Oblato; March 3, 2021.
- Battiste JD, Ikeguchi A, Woo S, Sharan S, Zhao YD, Cohoon A, et al. Phase Ib clinical trial of OKN-007 in recurrent malignant glioma. Journal of Clinical Oncology. Published online May 25, 2020. doi: 10.1200/JCO.2020.38.15_suppl.2538.