VORASIDENIB (VORANIGO)

Vorasidenib is a relatively new drug used to treat patients 12 years and older with grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation,   It is used after surgery, including biopsy, sub-total resection, or gross total resection, in  patients who are not in need of immediate chemotherapy or radiotherapy

Astrocytoma or oligodendroglioma with an IDH mutation is a type of brain tumour that starts in the glial cells of the brain and has a specific genetic mutation in the IDH1 or IDH2 genes. This mutation changes how the cells grow and can lead to an overproduction of a substance called 2-hydroxyglutarate (2-HG), which promotes tumour growth.  By blocking these proteins, vorasidenib, stops the abnormal production of 2 HG which helps to slow or stop the cancer from growing.

After successful trials, an early access scheme was introduced in the UK in 2024 and the drug has now been approved by The National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC) for use in the NHS, and many other countries including the US.

The most common side effects of the medicine can include an increased amount of liver enzymes, abdominal pain, diarrhoea, tiredness, and a decreased amount of blood platelets, although we have had many reports from patients who tolerate the drug very well with few side effects.   Vorasidenib may affect fertility in males and females – this is something you may want to discuss with your medical team.

The treatment comes in the form of one pill taken daily.  You may be subject to regular blood checks – twice monthly initially and then once monthly.

If you are interested in finding out more about this innovative treatment, we recommend you speak to your neuro-oncologist for more details and to find out if it is an option for you.