Vorasidenib (VORANIGO™) for brain tumours

What is vorasidenib?

Vorasidenib is a drug that inhibits the enzymes IDH1 and IDH2 and can cross the blood-brain barrier. Its ability to cross the blood-brain barrier makes it more effective at treating some tumour cells.

In healthy cells the enzymes IDH1 and IDH2 are important in ensuring cells in our body get the energy they need to grow and survive. They play an important role in lots of processes in the body. However, when these enzymes are mutated in astrocytoma or oligodendroglioma they cause the overproduction of 2-hydroxyglutarate (2-HG), which promotes tumour growth.

Vorasidenib has been developed by scientists to preferentially target astrocytoma and oligodendroglioma mutated forms of IDH1 and IDH2 to block the overproduction of 2-HG. This slows disease progression and delays the time to radiotherapy or chemotherapy and offers a new, targeted treatment option for those diagnosed with this type of tumour.

The phase 3 Investigating Vorasidenib in Glioma (“INDIGO”) clinical trial of the drug, which involved 331 patients from 10 countries, showed that vorasidenib significantly slowed tumour growth in some patients with grade 2 gliomas who had mutations in the IDH1 or IDH2 genes. Vorasidenib also significantly delayed time to next intervention (TTNI) compared to those who received a placebo instead of vorasidenib.

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Can I have vorasidenib for a brain tumour?

Vorasidenib is a treatment option for adult and paediatric patients over the age of 12 with a diagnosis of a grade 2 astrocytoma or oligodendroglioma with an IDH1 or IDH2 mutation, who are not in need of immediate chemotherapy or radiotherapy following surgery.

You can speak to your healthcare team, to understand whether vorasidenib would be a treatment option for you.

What happens if Vorasidenib is suitable?

Vorasidenib has been approved by NHS Scotland to treat people aged 12 or older with IDH-mutated, grade 2 gliomas – including astrocytomas and oligodendrogliomas. Guidance on medicine in Scotland is provided by the Scottish Medicines Consortium (SMC).

The National Institute for Health and Care Excellence (NICE) has recommended that vorasidenib is used for brain tumour treatment for eligible patients in England and Wales too. This means that patients will now be able to access vorasidenib via the NHS.

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What was The Brain Tumour Charity’s involvement with vorasidenib?

The Brain Tumour Charity has been involved throughout the NICE review of vorasidenib. Initially taking part in scoping for this appraisal and then submitting evidence from the community for the committee to consider.

The evidence from the community involved research drawn of lived experience. It also incorporates results from a survey and workshop involving the relevant patient population.We will continue our involvement to ensure that our community is represented.

While we have been involved throughout the NICE review, we have not funded any research directly into vorasidenib.

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