FDA Grants Approval to VORANIGO for New Brain Tumour Treatment Drug

University of Findlay
By Reid Rusholme
Thursday, 03 October, 2024


FDA Grants Approval to VORANIGO for New Brain Tumour Treatment Drug

The Food and Drug Administration (FDA) has recently approved a potentially groundbreaking treatment for gliomas. Until now, the treatment for gliomas, a type of brain tumor, has been limited to surgical removal; VORANIGO has changed that. Developed by French company Servier Pharmaceuticals, the drug offers hope to those with Grade 2 IDH-mutant glioma — a rare yet aggressive cancer with a current five-year survival rate of 40 to 50%. As a result, the development has been extensively welcomed by both the patients who are suffering and their healthcare providers alike.

Brain tumors present in a variety of ways, with different causes, symptoms, and outlooks. Unfortunately, they are no small issue; the National Foundation of Cancer Research estimates that 90,000 brain tumors are diagnosed every year in the US alone. While not all are cancerous, all brain cancers are tumors, and as with other cancers, tumors are often caused by mutations in DNA that can cause cells to grow uncontrollably. Among these cancers, grade 2 IDH-mutant gliomas are increasingly common. They originate in glial cells, which play a crucial role in supporting the function of other cells in the central nervous system. Gliomas can cause a whole host of issues ranging from headaches, nausea, memory loss, and personality changes to seizures and vision problems.

Up until now, the best treatment for tumors has been a combination of chemotherapy drugs and surgery to remove the tumor. The problem is glioma tumors often regrow. But what if there was a drug you could take after surgery to reduce the chances of regrowth? That is where VORANIGO comes in; it acts as an inhibitor of isocitrate dehydrogenase-1 (IDH1) and isocitrate dehydrogenase-2 (IDH2). These are enzymes that would usually help healthy cells break down nutrients for energy, but mutations can lead to cancer by preventing cells from developing properly — resulting in uncontrolled cell growth. VORANIGO works by reducing the activity of both these enzymes.

This system of regulating dangerous growth gives oncologists a new tool to combat cancers that have traditionally been treated with chemotherapy, radiation therapy, and, more recently, targeted chemotherapy. It is only through research and development that we can expand the toolbox, development that will only continue with programs such as the University of Findlay online PharmD program, which are essential in preparing the next generation of pharmacists to understand, manage, and dispense the growing array of cutting-edge treatments.

Patients and healthcare workers alike exponentially appreciate the approval of any new cancer drug, but this drug offers an entirely new way to treat debilitating tumors. As of writing, VORANIGO is available through a range of insurance providers in the US and has copay options. Further afield, regulatory bodies in Canada and the EU are reviewing applications for approval. Servier Pharmaceuticals is not resting on this achievement and has a range of cancer drugs in the pipeline; they are in the third phase of trials for Ivodsidenib, a drug targeting IDH1 mutations in leukemia. Trials are underway to see if VORANIGO’S efficacy can be improved when combined with other drugs, and Servier is in the earlier stages of trialing seven new treatments for tumors.

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