FDA Grants Oblato Brain Tumor Drug Rare Pediatric Disease Designation

Rare Daily Staff

The U.S. Food and Drug Administration has awarded Rare Pediatric Disease designation to Oblato’s OKN-007, an experimental brain cancer.

The FDA granted the designation to OKN-007 for diffuse intrinsic pontine glioma (DIPG), a fast-growing pediatric cancer that starts in the brain stem. It is one of several sub-categories of malignant gliomas, deadly cancers of the brain and spinal cord. It also granted Orphan Drug designation of OKN-007 for treatment of malignant glioma.

According to the National Brain Tumor Society, approximately 26,000 Americans will be diagnosed with primary malignant brain tumors this year. Of those, up to 300 will be cases of DIPG.

OKN-007 was discovered by Oklahoma Medical Research Foundation scientists Rheal Towner and Robert Floyd. Oblato acquired all rights to OKN-007 from the foundation, and the company is currently testing the investigational drug in a phase 2 clinical study of 56 patients suffering from recurrent glioblastoma, the most aggressive form of glioma. The patients are being treated with the drug in combination with another medication, temozolomide, at eight sites in the United States.

In pre-clinical studies, Towner has also shown that OKN-007 inhibits growth of human DIPG tumors implanted in experimental models. Oblato is planning to begin clinical trials in DIPG patients in 2021. “Right now, there is no effective treatment for this deadly brain cancer,” said Towner.

Going forward, Oblato and OMRF will continue collaborating, with a focus on improving treatment for patients suffering from a variety of solid-tumor cancers.

The FDA grants Rare Pediatric Disease designation for serious or life-threatening diseases with manifestations in individuals aged from birth to 18 years, including access to the FDA’s expedited review and approval process. The designation makes OKN-007 eligible for a Rare Pediatric Disease Priority Review voucher upon approval of the therapy by the FDA.

The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, Sarepta sold its priority review voucher to Vifor Pharma $111 million in February 2020.

Photo: Oklahoma Medical Research Foundation scientist Rheal Towner

Editor’s note: This story was updated to correct the most recent sales of a priority review voucher.