The MHRA has approved vorasidenib (Voranigo) for patients aged 12 and above with grade 2 astrocytoma or oligodendroglioma harbouring a susceptible IDH1 or IDH2 mutation. The treatment is intended for those not requiring immediate chemotherapy or radiotherapy following surgery.
These tumours originate in glial cells and are driven by mutations in IDH1 or IDH2 genes. The mutation leads to excess production of 2-hydroxyglutarate (2-HG), which promotes tumour growth. Vorasidenib works by blocking the proteins responsible for this abnormal production.
Approval was granted through Project Orbis, a global regulatory initiative led by the US FDA and involving agencies in Australia, Canada, Singapore, Switzerland, Brazil and Israel. The programme aims to accelerate access to promising cancer therapies.
Julian Beach, MHRA Executive Director, Healthcare Quality and Access, said: “Project Orbis opens access to safe and effective new cancer drugs for patients that need them, and we are assured that the appropriate regulatory standards for the approval of this medicine have been met. As with all products, we will keep the safety of this medicine under close review.”
Vorasidenib is taken orally once daily. Adults and children weighing 40kg or more should take 40mg daily, while those under 40kg are recommended to take 20mg daily.
The approval is backed by data from a phase 3 trial involving 331 patients. Those treated with vorasidenib showed significantly longer progression-free survival—27.7 months compared to 11.1 months for placebo.
Common side effects include elevated liver enzymes, abdominal pain, diarrhoea, fatigue and reduced blood platelets, affecting more than 1 in 10 patients.
