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The Food and Drug Administration (FDA) on Aug. 6 approved VORANIGO, the first targeted therapy for Grade 2 Isocitrate dehydrogenase (IDH) mutant glioma, a type of brain tumor.
Available to patients as a once-daily pill, VORANIGO is the first significant treatment breakthrough in nearly 25 years for Grade 2 IDH mutant glioma, the drug’s maker Servier explained.
“Servier is relentless in our commitment to bringing transformative new therapies to patients,” said David K. Lee, CEO of Servier Pharmaceuticals, a member of the Biotechnology Innovation Organization (BIO). “The approval of the first targeted therapy for patients living with IDH mutant glioma is evidence of this commitment in action.”
Stopping the growth of gliomas
“Gliomas are constantly growing, relentless and incurable brain tumors with inevitable disease progression that can lead to a range of debilitating symptoms for these patients,” Lee explained.
While these tumors can be categorized as either cancerous or noncancerous, they undoubtedly affect brain function in patients as they can press up against parts of the brain causing symptoms like headaches, seizures, nausea and vomiting, vision or hearing problems, and balance problems, like dizziness and trouble walking—among others.
Gliomas can also become cancerous, setting patients on an arduous clinical pathway that can lead to surgery, radiation therapy, and/or chemotherapy. Surgery of these gliomas can be notably difficult, especially if they are near delicate areas of the brain.
“Diffuse gliomas with IDH mutations represent the most common malignant primary brain tumors diagnosed in adults younger than 50 years of age,” said Servier. “They are not curable with current therapies and without treatment they continue to grow and infiltrate normal brain tissue.”
“Our mission is to deliver hope to a community of patients that haven’t seen treatment advances in more than two decades,” said Lee. “I’ve spoken with many patients living with this condition now. … They are in the prime of their lives, otherwise healthy, at the peak of their careers, often starting families, raising children and planning for their future. A glioma diagnosis is devastating and can impact all aspects of their life.”
How VORANIGO works
VORANIGO is “an isocitrate dehydrogenase-1 (IDH1) and isocitrate dehydrogenase-2 (IDH2) inhibitor, indicated for the treatment of adult and pediatric patients 12 years and older with Grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation following surgery including biopsy, sub-total resection, or gross total resection,” said Servier.
“In healthy human cells, a family of genes called isocitrate dehydrogenases (IDH) help break down nutrients and generate energy for cells,” Servier explained. However, mutations in the IDH1 and IDH2 genes can lead to a variety of cancers because these mutations prevent cells from developing (or differentiating) into the kind of healthy cells they are ultimately supposed to become. “When cells cannot differentiate properly, they may begin to grow out of control. In IDH-mutant gliomas, VORANIGO works by reducing the activity of the mutant IDH1 and IDH2 enzymes, to help control the disease,” according to Servier.
VORANIGO’s approval means that patients can treat gliomas after surgery or biopsy with something as simple as a once-daily pill, thus allowing them to stave off or prevent regrowth of glioma tumors.
“Patients living with Grade 2 IDH-mutant gliomas have long faced the harsh reality of an incurable disease with very limited post-surgery treatment options,” said Ralph DeVitto, President & CEO, of the American Brain Tumor Association. “The FDA approval of VORANIGO marks a monumental breakthrough in glioma treatment, offering renewed hope for patients and their families living with this relentless disease.”
“Today’s approval of VORANIGO is an enormous leap forward in cancer care, and a defining moment for people living with Grade 2 IDH-mutant glioma,” said Arjun H. Prasad, Chief Commercial Officer, Servier Pharmaceuticals. “VORANIGO, which is the first breakthrough in this specific disease area in nearly 25 years, offers patients unprecedented improvement in progression free survival. We are proud to deliver this first-of-its-kind therapy to patients in need, and we remain committed to bringing innovative targeted therapies to people with cancer.”
