Servier receives news that NICE draft guidance recommends vorasidenib (VORANIGO™q) for use in the NHS for eligible patients with low-grade glioma

• Vorasidenib is the first oral targeted therapy authorised for the treatment of adults and paediatric patients aged 12 years and older with Grade 2 IDH-mutant gliomas, who have undergone surgical resection and are not in immediate need of radiotherapy or chemotherapy¹

London, 31^st March 2026 – Today, Servier UK, entity of the Servier Group, an independent international pharmaceutical company governed by a foundation, announced that the National Institute for Health and Care Excellence (NICE) has published final draft guidance recommending vorasidenib (VORANIGO™q) a once-daily oral targeted therapy indicated for the treatment of Grade 2 astrocytoma or oligodendroglioma with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation or isocitrate dehydrogenase-2 (IDH2) mutation in adults and paediatric patients 12 years and older, who are not in need of immediate chemotherapy or radiotherapy following surgical intervention.¹ 

Based on NICE’s final draft guidance published on 31^st March,  recommending vorasidenib from today, immediate access is available to eligible patients across England and Wales via interim funding through the cancer drugs fund (CDF).

IDH mutant grade 2 gliomas frequently affect people with median age 20–40, and may cause a range of neurological symptoms, such as seizures.² With around 400 grade 2 glioma patients diagnosed each year in the UK,^3,4,5 these tumours are not curable and generally continue to grow if they are not treated. Until now, the standard of care has been surgery followed by active observation or, when needed, radiotherapy and chemotherapy.

Vorasidenib is the first and only approved therapy designed to target mutant IDH enzymes in grade 2 glioma and has the ability to cross the blood-brain barrier.⁶ The blood-brain barrier normally prevents most drugs from reaching the brain.  At diagnosis, patients are tested for the presence of an IDH1 or IDH2 mutation and the identification of this mutation means that they are potentially eligible for treatment with vorasidenib.

“We are so pleased that based on the NICE draft guidance, eligible people living with Grade 2 IDH-mutant glioma in England and Wales will from today be able to access vorasidenib through the NHS via interim funding through the CDF. This marks a significant milestone for patients, their families and their healthcare teams who have been waiting for new options to help slow disease progression for over 20 years.

Our focus has always been on ensuring that innovation translates into real-world access, so now we turn our attention to ensure this medicine is available to all eligible patients across the UK.” said Paula Valencia, General Manager, Servier UK.

The draft guidance is based on results from the pivotal INDIGO study, an international, double-blind, randomised, placebo-controlled, phase 3 trial, which assessed the efficacy and safety of vorasidenib therapy in patients with residual or recurrent grade 2 IDH-mutant glioma who had been treated with surgery only. The study demonstrated that vorasidenib increased the time to tumour progression (a median of 27.7 months [17.0-NE] for vorasidenib vs 11.1 months [11.0-13.7] for placebo) (HR, 0.39; 95% CI, 0.27 to 0.56; 1-sided p<0.001). The most common adverse reactions were increased ALT (59.3%), increased AST (45.5%), increased GGT (37.7%), fatigue (36.5%) and diarrhoea (24.6%)¹. The most common Grade ≥3 adverse events for patients receiving vorasidenib vs placebo were an increase in liver enzymes (ALT (9.6% vs 0) and AST (4.8% vs 0).^1,6   

‘Today’s draft guidance to recommend vorasidenib for use in the NHS is a hugely important moment for the brain tumour community. This decision will enable access to the first new therapy in over two decades, offering hope of delaying disease progression for eligible Grade 2 glioma patients and their families.

This is a powerful reminder of what can be achieved when patients, clinicians, and industry work together to drive change. We hope this marks the beginning of a new era of progress for everyone affected by brain tumours.” said Mary Burton & Dawn Emerton, Trustees, Astro Brain Tumour Fund.

In addition to this draft recommendation by NICE which makes the treatment available from today for eligible patients in England and Wales via interim funding through the CDF, vorasidenib has also been accepted for use in Scotland by the Scottish Medicines Consortium (SMC). This means that vorasidenib is now accepted for use in the NHS Scotland for the treatment of Grade 2 astrocytoma or oligodendroglioma with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation or isocitrate dehydrogenase-2 (IDH2) mutation in adults and paediatric patients 12 years and older, who are not in need of immediate chemotherapy or radiotherapy following surgical intervention.

About the INDIGO Phase 3 Trial (NCT04164901)⁶

The INDIGO study included 331 adults and adolescents ≥ 12 years old weighing ≥ 40 kg. The primary end point was progression free survival (PFS) which was measured by a blinded independent review committee (BIRC) and demonstrated that vorasidenib increased the time to tumour progression (a median of 27.7 months [17.0-NE]  for vorasidenib vs 11.1 months [11.0-13.7]  for placebo) (HR, 0.39; 95% CI, 0.27 to 0.56; 1-sided p<0.001).  A key secondary end point was the time to the next anticancer intervention, also known as time to next intervention (TTNI). Other secondary end points were objective response rate, tumour growth rate by volume, safety and overall survival for which follow-up is still ongoing.

Crossover from placebo to vorasidenib was permitted upon confirmation of imaging-based disease progression. Safety outcomes were also evaluated throughout the study.

INDIGO met its major efficacy outcome of PFS and key secondary endpoint of TTNI at the prespecified second interim analysis. The primary outcome, PFS was statistically significant and clinically meaningful in favour of the vorasidenib arm. TTNI was also statistically significant (HR, 0.26; 95% CI, 0.15 to 0.43; 1-sided p<0.001). Median TTNI was not reached for vorasidenib and was 17.8 months for placebo. Vorasidenib was also shown to reduce the tumour volume by a mean of 2.5% (TGR of –2.5%; 95% CI: -4.7% to -0.2%) every 6 months, while tumour volume increased by a mean of 13.9% (TGR of 13.9%; 95% CI: 11.1% to 16.8%) every 6 months for patients randomised to the placebo arm, as measured by a BIRC. Adverse events of grade 3 or higher occurred in 22.8% of the patients who received vorasidenib and in 13.5% of those who received placebo. An increased alanine aminotransferase level of grade 3 or higher occurred in 9.6% of the patients who received vorasidenib and in no patients who received placebo.

About Glioma⁷

Gliomas are tumours that arise from glial or precursor cells within the brain and the central nervous system (CNS). Diffuse gliomas are characterised by their poorly defined boundaries, meaning they infiltrate healthy tissue rather than forming a distinct mass. The World Health Organization (WHO), sub-divides and grades adult-type diffuse gliomas into three categories based on molecular testing to identify the presence (or lack thereof) of mutations in the metabolic enzyme isocitrate dehydrogenase (IDH), and other molecular markers: ⁷  

• Astrocytoma, IDH-mutant (CNS WHO grades 2-4)  
• Oligodendroglioma, IDH-mutant and1p19q-codeleted (CNS WHO grades 2-3)  
• Glioblastoma, IDH-wildtype (CNS WHO grade 4) 

Grading is from 1-4 and is a system used by pathologists to assess how closely cancer cells resemble normal cells under a microscope. Grades 1 and 2 are considered to be low-grade gliomas.

About vorasidenib¹

Vorasidenib is an oral targeted therapy for the treatment of adult patients and paediatric patients aged 12 years and older with Grade 2 IDH-mutant gliomas, who have undergone surgical resection and are not in immediate need of radiotherapy or chemotherapy. Vorasidenib directly inhibits mIDH1/2 enzymes to block the abnormal production of 2-hydroxyglutarate (2-HG), a known driver of tumour development. Vorasidenib crosses the blood-brain barrier and penetrates the tumour, helping to slow the progression of the disease.

Product information is available here: Voranigo 40 mg Film Coated tablet - Summary of Product Characteristics (SmPC) - (emc) | 101421

About Servier in the UK

Servier is an independent research foundation with no shareholders and as such, revenue from the sales of its medicines is directly invested back into the research and development of our future treatments.

Servier is a leader in IDH-mutant targeted therapies and devotes more than 65% of its research and development budget to Oncology. Servier aspires to advance more targeted therapies by identifying mutations and understanding how these mutations impact cancer and its progression. Servier believes it can serve more people by helping the right patients find the right treatment, at the right time.

For more information, visit: www.servier.co.uk