The Medicines and Healthcare products Regulatory Agency (MHRA)
has today, 16th September 2025, approved the medicine vorasidenib
(Voranigo) to treat adults and children 12 years of age and older
with certain types of brain tumours, known as grade 2 astrocytoma
or oligodendroglioma with a susceptible isocitrate dehydrogenase
mutation (IDH1 or IDH2).
The medicine is for patients who are not in need of immediate
chemotherapy or radiotherapy following surgical
intervention
Astrocytoma or oligodendroglioma with an IDH mutation is a type
of brain tumour that starts in the glial cells of the brain and
has a specific genetic mutation in the IDH1 or IDH2 genes. This
mutation changes how the cells grow and can lead to an
overproduction of a substance called 2-hydroxyglutarate (2-HG),
which promotes tumour growth.
By blocking these proteins, vorasidenib, stops the abnormal
production of 2 HG which helps to slow or stop the cancer from
growing.
Vorasidenib has been approved through Project Orbis, a global
partnership between the MHRA, and its regulatory counterparts in
Australia, Canada, Singapore, Switzerland, Brazil, and Israel,
coordinated by the US Food and Drug Administration (FDA). The
programme reviews and approves promising cancer drugs, helping
patients to access treatments more quickly.
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 as oral tablets once daily. The recommended
dosage for adults is 40mg once daily, while for children aged 12
years and older, the dose is based on body weight. Patients
weighing 40kg or more are recommended to take 40mg once daily,
while those weighing less than 40kg should take 20mg once
daily.
This approval is supported by evidence from a randomised,
double-blind clinical trial involving 331 patients with IDH1- or
IDH2-mutant grade 2 astrocytoma or oligodendroglioma. Patients
receiving vorasidenib had significantly longer progression-free
survival (27.7 months versus 11.1 months for placebo).
The most common side effects of the medicine (which may affect
more than 1 in 10 people) include an increased amount of liver
enzymes, abdominal pain, diarrhoea, tiredness, and a decreased
amount of blood platelets.
As with any medicine, the MHRA will keep the safety of
vorasidenib under close review.
Anyone who suspects they are having a side effect from a medicine
are encouraged to talk to their doctor or another healthcare
professional and report it directly to the Yellow Card scheme,
either through the website (https://yellowcard.mhra.gov.uk/)
or by searching the Google Play or Apple App stores for MHRA
Yellow Card.
Notes to editors
-
The new marketing authorisation was granted on 16th September
2025, to Les Laboratoires Servier
-
More information can be found in the Summary of Product
Characteristics and Patient Information leaflets which will
be published on the MHRA
Products website within 7 days of approval.
-
The aim of Project Orbis is to deliver faster patient access
to innovative cancer treatments with potential benefits over
existing therapies. For more information, see: Project Orbis -
GOV.UK
