Servier receives UK Marketing Authorisation for vorasidenib (VORANIGO™q) for low grade glioma

  • Vorasidenib is the first and only 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 chemotherapy.1

London, September 16th 2025 – Today, Servier announced that the Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing authorisation in the UK for (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

These 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, radiation 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 where they have a therapeutic effect.

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.

The marketing authorisation 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 11.1months [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  

“This approval is an important milestone for the low grade glioma brain tumour community. For too long, patients with Grade 2 gliomas have had limited options. This new treatment option, a first in two decades, offers patients a chance to delay disease progression.” said Mary Burton & Dawn Emerton, Trustees, Astro Brain Tumour Fund.

“Patients diagnosed with Grade 2 IDH-mutant gliomas—many of whom are young and otherwise healthy —have had only limited treatment options after surgery such as radiotherapy and chemotherapy. Vorasidenib offers a new treatment option for suitable patients with Grade 2 IDH-mutant gliomas, addressing an important unmet need.” said Liam Welsh, MA PhD MBBS MSc MRCP FRCR, Consultant Clinical Oncologist, Neuro-oncology Unit, The Royal Marsden NHS Foundation Trust.

Following marketing authorisation, the National Institute for Health and Care Excellence (NICE) and Scottish Medicines Consortium (SMC) submissions are under review for the reimbursement of vorasidenib via the National Health Service (NHS).

“Bringing the first targeted therapy in over 20 years to the UK is an important step forward for people living with Grade 2 IDH-mutant glioma. It reflects years of research and collaboration, and offers patients, many in their 30s and 40s, a new option to slow disease progression.”

“Access is a priority, and we are committed to working closely with the government and health authorities so that we can bring this new medicine to eligible patients in the UK” said Paula Valencia, General Manager, Servier UK.

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, slowing the progression of the disease. Vorasidenib is licensed in United States, Canada and 3 other countries worldwide. (SPC attached)

About Servier

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

Media Contact (UK):

AXON Team
Daisy Bartlett

AXONServierUKTeam@axon-com.com
+44 7909 985089

  1. VORANIGO® SmPC.
  2. Pan Z, Bao J, Wei S.  Front Oncol. 2025 Jun 27;15:1628195. doi: 10.3389/fonc.2025.1628195. PMID: 40657255; PMCID: PMC12245911.
  3. NICE TA23. Guidance on the use of temozolomide for the treatment of recurrent malignant glioma (brain cancer). Last accessed September 2025.
  4. Weidl et al J Neurooncol. (2025) 174:391–400.
  5. Bauchet et al Neurochirurgie. 2025; volume 71, Issue 3, 101627.
  6. Mellinghoff IK et al. N Engl J Med. 2023;389:589-601.
  7. Louis D et al. Neuro Oncol. 2021;23:1231-1251.

M-VOR-UK-00013

Date of Preparation September 2025

19/09/2025