NICE backs Novartis duo for NHS use in young glioma patients


An oral therapy that can allow children and adolescents with an aggressive form of brain cancer to be treated at home rather than in hospital has been recommended for use by the NHS in England and Wales.

The combination of Novartis’ Finlee (dabrafenib) and Spexotras (trametinib) is now recommended by NICE for paediatric patients with relapsed/refractory BRAF V600E-positive glioma, within three months of their approval for this type of cancer in the UK.

The new guidance covers low-grade glioma (LGG) as an option for initial treatment, as well as fast-growing high-grade glioma (HGG) patients after at least one radiation or chemotherapy treatment. The Finlee/Spexotras regimen is the first standard of care for the latter group, who have a long-term survival rate of less than 10%.

Gliomas are the most common type of brain cancer in children and young adults, and the BRAF V600E mutation is present in 15%-20% of paediatric LGGs and in 5%-10% of HGGs, according to Novartis. NICE estimates that around 30 children in England could be eligible for treatment with the new combination therapy.

The two drugs were licensed by the MHRA thanks to the phase 2/3 TADPOLE clinical trial carried out at Great Ormond Street Hospital (GOSH) in London, which showed that the regimen stalled tumour growth in people with LGG for an average of over two years – approximately three times longer than current drugs.

In HGG patients, more than half (56%) of children responded to treatment, with a median duration of response of 22 months and a median overall survival (OS) of 33 months. For comparison, the response rate, with conventional second-line therapy is typically 12% with a median OS of less than six months.

The recommendation has been celebrated by The Brain Tumour Charity, which sent representatives to provide evidence on behalf of glioma patients to NICE’s appraisal committee.

“We are delighted that NICE has approved the first new treatment for paediatric brain tumours in decades,” said Dr Michele Afif, chief executive of the charity. “Though this will only affect a small population, it’s of huge significance to them and their loved ones and represents real progress.”

The Scottish Medicines Consortium (SMC) is currently considering use of Finlee/Spexotras for this indication, and Novartis said it is working with the agency to try to ensure equitable access across the UK.

The company said NICE is the first European health technology assessment (HTA) organisation to complete a review of the two drugs in this setting.

Meanwhile, another drug for glioma may soon be on the way in the US and Europe, after Servier filed for approval of its vorasidenib candidate for IDH-mutated LGG. IDH mutations are commonly seen in gliomas and are present in somewhere between 50% and 81% of cases.

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