Novartis’ brain tumour drug Votubia launched in UK
pharmafile | September 29, 2011 | News story | Sales and Marketing | Afinitor, Votubia
Novartis has launched its orphan drug Votubia in the UK for patients with a rare type of brain tumour.
Votubia (everolimus) is licensed for patients over three years of age with subependymal giant cell astrocytoma (SEGA) – a benign brain tumour – associated with tuberous sclerosis complex whose tumours cannot be removed.
Votubia, taken as a once-daily tablet, was shown to be effective at treating patients with SEGA by shrinking the volume of the main brain tumours.
In a recent Phase III study the main brain tumour shrank by half in around 30% of patients and by about a third in 70% of patients.
Novartis said its drug provides an alternative for patients who previously faced invasive brain surgery as the only treatment option for growing SEGAs.
Because the number of patients with tuberous sclerosis is very rare – with around 8,000 cases in the UK – Votubia was granted orphan status by the European Medicines Agency.
But the EMA’s approval is conditional upon follow up studies from Novartis, which are designed to show the long-term effects of the medicine and how it is handled in the body.
Chris Kingswood, head of research at the Tuberous Sclerosis Association, said: “For a long time there has been a desperate need for a useful alternative treatment to invasive brain surgery.
“Everolimus is the first licensed product we can offer patients to attack the cause of their debilitating condition; it works by blocking a protein that acts as an important regulator of tumour cell growth.
“The way it works in the signalling pathway can be simply likened to blocking a receiver so it cannot process signals from the aerial on an analogue radio, so that the signals can no longer transmit to drive in this case tumour cell growth.”
Novel mode of action
SEGAs primarily affect children and adolescents and may pose a significant medical risk, including the potential for increased intracranial pressure, swelling in the brain (hydrocephalus), neurological defects and seizures.
Votubia targets mTOR, a protein that acts as an important regulator of tumour cell division, blood vessel growth and cell metabolism.
Tuberous sclerosis complex (TSC) is caused by defects in two particular genes – when these genes are defective mTOR activity is increased, which can cause uncontrolled tumour cell growth, leading to the formation of benign tumours throughout the body, including the brain.
By inhibiting mTOR activity in this signalling pathway, Votubia may reduce cell proliferation, blood vessel growth and glucose uptake related to SEGA associated with TSC.
Everolimus is already licensed to treat a range of conditions in Europe, with the biggest proportion of its sales coming under the Afinitor brand for kidney cancer.
The cost of Votubia will be £75 per 5mg oral dose, according to Novartis.
Ben Adams
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