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Mateon Therapeutics' brain tumor drug granted US FDA rare pediatric disease tag

Mateon Therapeutics Inc.'s experimental brain tumor therapy has been granted rare pediatric disease designation by the U.S. Food and Drug Administration.

OT101, or trabedersen, an RNA therapeutic that works by blocking immunosuppression in the tumor, is being developed to treat diffuse intrinsic pontine glioma, or DIPG, Mateon said.

DIPG is the most common brain stem tumor in children and has no current standard of care; chemotherapy and radiation treatments have not shown effect in extending patients' survival. The mean survival rate is nine to 12 months from the time of diagnosis, according to Mateon.

If South San Francisco, Calif.-based Mateon receives FDA approval for the drug to treat this type of brain tumor, the biotechnology company will receive a priority review voucher under the program, Mateon said in its Sept. 23 press release.

OT101 was previously granted orphan-drug designation by the FDA. In a phase 2 trial evaluating the therapy in adults with a fast-growing brain tumor known as high-grade glioma, OT101 was an effective single treatment for patients, including those with glioblastoma multiforme, the most aggressive form of brain tumor.