At The Brain Tumour Charity, we share the disappointment many in our community have felt following the draft guidance from the National Institute for Health and Care Excellence (NICE) not to recommend vorasidenib for routine NHS use in England.
Vorasidenib is a targeted oral therapy developed for people aged 12 and over with low-grade, IDH-mutant gliomas. It has shown potential to delay the need for radiotherapy or chemotherapy treatments – which, while effective, can have long-term impacts on quality of life.
Clinical trials suggest it can extend the time before further intervention is needed, potentially preserving quality of life for longer. Given the limited options currently available, it could represent meaningful progress for eligible patients.
This marked a significant moment of hope for families affected by a brain tumour diagnosis. While the treatment has been authorised by the MHRA, it is not yet available on the NHS. That will depend on a final decision from NICE.
Where things stand
NICE began reviewing vorasidenib through its Single Technology Appraisal process in November 2024. A NICE appraisal committee met in September 2025 to consider the available evidence. In October, it issued a draft recommendation not to make the treatment available on the NHS.
This decision is still under consultation. Until then, there is a vital opportunity for further evidence and community insight to help shape the final decision.
That’s why we need to hear from you. By sharing your views, experiences or reflections — whether through our short survey or by sending us a message — you can help ensure the voices of those affected are at the heart of the process.
While we recognise the purpose behind the UK’s specific regulatory arrangements, it is more than disappointing to see how decisions like these – even in draft – create yet another barrier to accessing new treatments for patients whose access to promising advances has been minimal and unmet need painfully high. The Brain Tumour Charity continues to campaign for a regulatory system that is flexible, responsive to the unique challenges people diagnosed with a brain tumour face every day and open to reviewing decisions so that they can access promising new treatments without further delays.
Dr Michele Afif – CEO of The Brain Tumour Charity
What we’re doing — and how you can help
We remain committed to advocating for equitable access to innovative treatments like vorasidenib, which offer real progress for people living with brain tumours. This is not just about approval — it’s about ensuring that those who could benefit most from this treatment are able to access it, without delay.
As part of our response to NICE, we are:
- Collecting feedback from our community through a short survey and personal testimonies
- Sharing lived experiences directly with NICE
- Engaging with clinicians, policymakers and media to raise awareness
- Encouraging exploration of all possible routes to access, in partnership with others
It is not uncommon for new treatments not to be recommended at this stage of NICE’s process. We know that around 84% go on to be approved after further review.
Your input could help shape the final outcome.
We need your voice
Whether you are a patient, family member, carer or clinician, your insight matters.
Please complete our short survey to share your views on the draft decision and what access to a treatment like vorasidenib could mean for you or your loved ones.
We’re also inviting personal messages, voice notes or short video statements. These may be used (with permission) to support our submission and wider campaigning.
To share your message, email policy@thebraintumourcharity.org
Why this moment matters
This decision highlights the broader challenges of bringing new treatments to people affected by brain tumours — challenges that are made more urgent by the lack of existing options. Outcomes for brain tumour patients remain among the poorest of any cancer, and the need for more effective, less harmful treatments is clear.
Vorasidenib is a meaningful development for a specific group of patients who have had few options for too long.
As globally the largest dedicated funder of research into primary brain tumours, The Brain Tumour Charity has committed over £60 million since 2015 in driving forward new treatments and improving quality of life. We will continue to stand with our community and push for decisions that reflect both the clinical evidence and people’s lived experience.
Stay informed and involved
- Read our FAQs below about the NICE appraisal and vorasidenib
- Complete the community survey to share your views
- Contact us at policy@thebraintumourcharity.org to share your story or ask a question
Together, we can ensure that the final decision reflects what matters most to those it will affect.
FAQs
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Vorasidenib is a drug that inhibits the enzymes IDH1 and IDH2 and can cross the blood-brain barrier.
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 therefore, slowing disease progression delaying time to chemotherapy and offering a new, kinder treatment option for those diagnosed with this type of tumour.
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The phase 3 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 not receiving vorasidenib (placebo).
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Patients with low grade astrocytoma or oligodendrogliomas who are not in need of immediate chemotherapy or radiotherapy following surgical intervention with the appropriate mutations.
If approved, the drug would be available to treat patients 12 years and older.
– https://www.cancer.gov/news-events/cancer-currents-blog/2023/vorasidenib-low-grade-glioma-idh-mutations
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Vorasidenib, was developed by scientists to cross the blood brain barrier and preferentially inhibit mutated IDH1 and IDH2 enzymes blocking the overproduction of a promoter of tumour growth, 2-HG. It is usually taken once a day as a tablet.
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Draft guidance of no from NICE means that, as the guidelines currently stand, vorasidenib will not be made available for use in the NHS.
NICE has determined that vorasidenib is not required to be funded and should not be used routinely in the NHS in England for the condition and population in the recommendations.
This is because the available evidence does not suggest that vorasidenib is value for money in this population.
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We would recommend you speak with your medical team about whether you are eligible for this treatment through a clinical trial.
We are currently aware of one open clinical trial (more information on question 12) you may want to discuss with your clinical team. – https://bepartofresearch.nihr.ac.uk/trial-details/trial-detail
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The draft guidance does not impact those already accessing vorasidenib who will still be able to access it as part of their treatment until they or their medical team consider it appropriate to stop.
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A draft ‘no’ does not mean the treatment will never be available on the NHS. It means that there are unanswered questions which need to be addressed before NICE can approve it. NICE has now released a Consultation Comment Document setting out the reasons behind the ‘draft no’ and we will be listening to the community and feeding that voice back into NICE.
This is a provisional decision which allows for consultation where feedback is considered before NICE makes a final decision.
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NICE have stated that they feel that there are uncertainties in the economic modelling, including assumptions on:
- how long people who had vorasidenib or placebo wait before starting a new treatment after their cancer gets worse
- if vorasidenib extends how long people live
- the quality of life of people in the model
Because of the uncertainties in the economic model, NICE have decided it is not possible to determine the most likely cost-effectiveness for vorasidenib.
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NICE have issued the draft guidance and will now seek comments on this guidance, which could turn the draft ‘no’ into a final approval. Once NICE has gathered views from all stakeholders, it will meet again to consider its decision. The date for this meeting will take place on the 20th November.
The Brain Tumour Charity has until Tuesday 4th November to submit a consultation response.
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We don’t know exact timings, but we hope to find out the decision either just before or just after Christmas 2025.
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USA: Vorasidenib was granted fast tracked designation by the FDA in the US in March 2023 and in February 2024 it granted priority review. On 6th August 2024 it was approved by the FDA for use in the US.
EU: In 2023, vorasidenib was given orphan designation by the European Medicines Agency (EMA).
Orphan medicines are used for the diagnosis, prevention and treatment of rare diseases and this means they are eligible for conditional marketing authorisation to ensure medicines are fast tracked to approval as they fulfil an unmet medical need.
In some cases, designated orphan medicines may be given to patients under compassionate use, a treatment option that allows the use of an unauthorised medicine outside a clinical study.
In February 2024, the EMA accepted vorasidenib for accelerated assessment which means evaluating a marketing authorisation application can take 150 days compared to the 210 days which is standard.
The European Commission issued marketing authorisation for vorasidenib on 17 September 2025.
Vorasidenib was given marketing authorisation in the UK in September 2025.
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The recent INDIGO clinical trial has ended its recruitment, and the results are published here and explained on our website here.
There is currently an open clinical trial exploring the safety of vorasidenib in combination with temozolomide. To find out more information about this trial please click below where there is information on exclusion criteria, contact information for the study team and locations. The trial is taking place at The Christie Hospital, Manchester and The Royal Marsden in Sutton and we advise speaking to your medical team regarding access and eligibility to this trial or other clinical trials.
https://bepartofresearch.nihr.ac.uk/trial-details/trial-detail
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The Brain Tumour Charity has been involved throughout this process, initially taking part in scoping for this appraisal and then submitting evidence from the community for the committee to consider.
This information was drawn from desk research of lived experience, but also from a survey and workshop involving the relevant patient population.
We will continue to be involved in this process and ensure that our community is adequately represented.
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This draft decision impacts patients in Wales and NI with NICE guidance applying for Wales and being generally adopted in NI.
However, in Scotland, the Scottish Medicines Consortium (SMC) is responsible for conducting Health Technology Assessments (HTAs) for new medicines – determining the clinical and cost effectiveness of potential new medicines, in a separate process to the NICE review.
We are aware that the manufacturer intends to follow this process, but we do not have a timeframe for this confirmed.
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We have not funded any research directly into vorasidenib.
References
- Cohen, A.,Holmen S., and Colman, H. (2013). ‘IDH1 and IDH2 Mutations in Gliomas’, Current Neurology and Neuroscience Reports. 13(5):345. Access: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4109985/
- Vorasidenib for treating astrocytoma or oligodendroglioma with IDH1 or IDH2 mutations after surgery in people 12 years and over TS ID 11974 https://www.nice.org.uk/guidance/awaiting-development/gid-ta11498 (last accessed 21.05.24)
- FDA approves new therapy for glioma patients for first time in decades: https://www.dana-farber.org/newsroom/news-releases/2024/fda-approves-new-therapy-for-glioma-patients-for-first-time-in-decades%22%EF%B7%9FHYPERLINK%20%22https://www.dana-farber.org/newsroom/news-releases/2024/fda-approves-new-therapy-for-glioma-patients-for-first-time-in-decades“ (last accessed 22.08.24)
The National Institute for Health and Care Excellence (NICE) has issued draft guidance that does not recommend vorasidenib as a new treatment on the NHS for grade 2 astrocytoma or oligodendroglioma with a susceptible isocitrate dehydrogenase mutation (IDH1 or IDH1).
