The Medicines & Healthcare Regulatory Agency (MHRA) has approved vorasidenib (Voranigo) for the treatment of adults and children aged 12 and older with specific types of brain tumors, explicitly grade 2 astrocytoma or oligodendroglioma with an IDH1 or IDH2 mutation. This new selection is intended for patients who do not require immediate chemotherapy or radiotherapy after surgery.
Astrocytoma and oligodendroglioma are brain tumors that start in glial cells. When they carry an IDH mutation, the cells produce an excess of a substance called 2-hydroxyglutarate (2-HG), which triggers tumor growth. Vorasidenib works by hindering this process, helping to slow or stop tumor progression.
The approval was approved through Project Orbis, an international programme coordinated by the U.S. FDA and involving regulators from the UK, Australia, Canada, Singapore, Switzerland, Brazil, and Israel. The initiative accelerates access to promising cancer treatments while ensuring strict safety standards are met.
Clinical evidence from a study of 331 patients showed that vorasidenib significantly extended progression-free survival 27.7 months compared with 11.1 months for patients on placebo. The medicine is taken once daily as tablets, with dosage based on age and body weight.
The most common side effects include raised liver enzymes, abdominal pain, diarrhea, tiredness, and low platelet counts. As with all medicines, the MHRA will continue to closely monitor its safety. Patients and healthcare professionals are encouraged to report suspected side effects through the Yellow Card scheme.
