Servier has received marketing authorisation from the Medicines and Healthcare products Regulatory Agency (MHRA) in the UK for vorasidenib (VORANIGO™q), a once-daily oral targeted therapy for adults and paediatric patients aged 12 years and older with Grade 2 IDH-mutant gliomas. The approval marks the first time a therapy designed to target mutant IDH enzymes has been authorised for this patient group.

Grade 2 gliomas, including astrocytomas and oligodendrogliomas, typically affect people in their twenties to forties and can cause neurological symptoms such as seizures. Until now, treatment options have been limited to surgery followed by active observation, with radiotherapy or chemotherapy reserved for later stages. The tumours are generally not curable and continue to grow if untreated.

Vorasidenib works by targeting IDH1 and IDH2 mutations, key drivers of tumour growth, and has the ability to cross the blood-brain barrier to reach the tumour. Patients are tested for these mutations at diagnosis to determine eligibility for treatment.

The approval is based on results from the pivotal INDIGO trial, an international, double-blind, randomised, placebo-controlled phase 3 study in patients with residual or recurrent Grade 2 IDH-mutant gliomas following surgery. The trial demonstrated a median progression-free survival of 27.7 months for patients receiving vorasidenib versus 11.1 months for placebo, representing a significant delay in tumour progression. Time to next anticancer intervention was also prolonged. Common side effects included elevated liver enzymes, fatigue, and diarrhoea, with serious liver enzyme elevations occurring in fewer than 10% of patients.

“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,” said Mary Burton, trustee of the Astro Brain Tumour Fund.

Liam Welsh, consultant clinical oncologist at The Royal Marsden NHS Foundation Trust, added: “Vorasidenib offers a new treatment option for suitable patients with Grade 2 IDH-mutant gliomas, addressing an important unmet need.”

Servier said submissions to the National Institute for Health and Care Excellence (NICE) and Scottish Medicines Consortium (SMC) are under review for reimbursement via the NHS. Paula Valencia, general manager of Servier UK, said: “Access is a priority, and we are committed to working closely with the government and health authorities to bring this new medicine to eligible patients in the UK.”

The INDIGO trial included 331 adults and adolescents, and safety outcomes were monitored throughout. Vorasidenib reduced tumour growth and prolonged the time to next intervention, making it the first therapy in over two decades to offer a targeted option for patients with low grade IDH-mutant gliomas.