The Ministery of Food and Drug Safety said it approved an orphan drug to treat glioma, a type of brain tumor. Glioma is a brain tumor that arises from glial cells in the brain and spinal cord.
The newly approved therapy is Voranigo (ingredient name Vorasidenib Citrate), developed by the French pharmaceutical company Servier. The drug was approved in two dose strengths, 10 mg and 40 mg.
It is used for patients age 12 or older and weighing 40 kg or more who have residual tumor after surgery and have grade 2 astrocytoma or oligodendroglioma with mutations in the IDH 1 or IDH 2 enzymes. Astrocytoma and oligodendroglioma are representative types of glioma that arise from glial cells.
IDH-mutant glioma is a malignant brain tumor caused by mutations in a specific gene (IDH) and is known to be the most common malignant brain tumor among younger adults under 50.
Voranigo is effective only in patients with mutations in the IDH 1 or 2 enzymes. When a mutation occurs, the body produces more than normal of the carcinogen 2-HG, causing tumors to grow rapidly, but Voranigo inhibits this process and blocks the growth of tumor cells. Because of this mechanism of action, it is called a "targeted therapy."
With this approval, new treatment opportunities are expected to open for brain tumor patients with IDH mutations. The Ministery of Food and Drug Safety said, "We will continue to do our best to quickly supply therapies whose safety and efficacy have been sufficiently verified, so that patients with rare and intractable diseases can have expanded access to treatment."