Today, the Scottish Medicines Consortium (SMC) have approved vorasidenib – also known by its brand name Voranigo – to be used by NHS Scotland to treat people aged 12 or older with IDH-mutated, low grade gliomas – including astrocytomas and ogliodendrogliomas.
We wholeheartedly welcome this news as it’s the first targeted drug to be approved to treat these tumours and only the second new drug to be approved to treat brain tumours since temozolomide in 2006.
Vorasidenib has been shown in a clinical trial to slow tumour growth and significantly delay the need for somebody to have chemotherapy or radiotherapy. This can be hugely beneficial because vorasidenib has also been shown to have much less of a negative impact on quality of life.
While this decision only affects people being treated in Scotland, we sincerely hope that this breakthrough treatment will soon become available in England, Northern Ireland and Wales too.
We asked Brooke, a student from North Lanarkshire who’s living with a brain tumour, to comment on the decision:
“I’ll be first in the queue to speak to my medical team now that vorasidenib is available on the NHS. It would be a massive relief to take the medication because I worry a lot about the tumour. It’s always in the back of my mind that one day it might… I find it really difficult to even say the words.
“What if it was to grow back or what if I was to go up a grade? Even to have to go down the road of having chemotherapy or radiotherapy fills me with dread, because I’ve been warned that that’s going to happen.”
What does this decision mean for the brain tumour community in Scotland?
Now that the SMC has advised that vorasidenib should be used to treat these tumours, NHS Scotland Health Boards are expected to consider that advice and make the medicine (or an equivalent) available locally.
Unfortunately, even when multiple Health Boards decide to make a new treatment available, variations in timings and pathway integration can occur. This means vorasidenib may become available through some Health Boards before others.
What does this mean for the brain tumour community in England, Northern Ireland and Wales?
Vorasidenib remains under review by the National Institute for Health and Care Excellence (NICE) for use in NHS England. They’re currently expected to publish their decision by 29 April. If approved by NICE, vorasidenib would become available to appropriate patients in England and Wales – the decision is also generally adopted in Northern Ireland.
We remain optimistic of a positive outcome from the NICE review for the brain tumour community, especially now that the treatment has been approved by the SMC.
What are we going to do next?
The Brain Tumour Charity and its Scottish Steering Committee will be carefully monitoring the roll out of vorasidenib across the 14 regional Health Boards in Scotland.
We’re keen to continue campaigning to make sure there is equitable access to the treatment across Scotland and eventually the whole of the UK.
As such, we’re also paying close attention to the result of the NICE review and will be ready to act when the results are published.
Our CEO Dr Michele Afif said: “This is a moment of real celebration for the brain tumour community in Scotland. A moment we hope to see repeated in England, Wales and Northern Ireland.
“Approvals for new treatments to be used within the NHS are all too rare in the brain tumour setting. This decision is a testament to the hard work of many stakeholders including researchers, clinicians, industry, charities and those with lived experience.”
Frequently asked questions
People in Scotland aged 12 years and older with low grade gliomas – including astrocytoma or oligodendrogliomas – who aren’t in need of immediate chemotherapy or radiotherapy following surgical intervention with the appropriate IDH-mutations.
The SMC is an independent body that makes decisions about whether new medicines can be prescribed routinely in Scotland. This means the SMC’s decision won’t change the availability of vorasidenib for people under the care of the NHS in England, Wales and Northern Ireland.
Vorasidenib remains under review by the National Institute for Health and Care Excellence (NICE) for use in NHS England. They’re currently expected to publish their decision by 29 April.
If approved by NICE, vorasidenib would become available to appropriate patients in England and Wales – the decision is also generally adopted in Northern Ireland.
Vorasidenib – also know by its brand name also known as its brand name Voranigo – is a drug that has been shown to effectively slow tumour growth in some types of low grade glioma.
When certain low grade gliomas – including astrocytoma or oligodendroglioma – have an IDH-mutation, it causes an overproduction of a molecule that promotes tumour growth.
Vorasidenib has been developed by scientists to block the overproduction of this molecule and slow tumour growth. In clinical trials, it was shown to significantly slow tumour growth and delay the need for chemotherapy or radiotherapy.
This is important as these treatments can have much more of negative impact on people than vorasidenib – meaning it’s a kinder but effective way to slow tumour growth.
It can also be taken as a tablet at home, whereas chemotherapy and radiotherapy can often require out-patient appointments at hospital.
If you live in Scotland, we recommend speaking with your medical team about whether you’re eligible for this treatment.
If you live in England, Northern Ireland or Wales, we recommend speaking with your medical team about whether you’re eligible for this treatment through a clinical trial.
The recent INDIGO clinical trial has ended its recruitment and the results are explained on our website here.
There is also currently a clinical trial exploring the safety of vorasidenib in combination with temozolomide, but recruitment has now closed.
The Brain Tumour Charity has been involved throughout this process, 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 people affected by the tumours that vorasidenib can treat.
We both submitted evidence and attended the Patient and Clinician Engagement (PACE) meeting that was part of the SMC review.
We’ll continue to be involved in this process as vorasidenib is reviewed for use in England, Northern Ireland and Wales. As well as working to make sure that this new treatment is made available in an equitable fashion throughout the UK.
We haven’t funded any research directly into vorasidenib, although we’ve been significantly involved in campaigning for it to be made availabe across the UK.