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23117411000001106

New Medicines

Tafinlar BRAF V600 mutation positive low grade glioma (LGG) or high grade glioma (HGG) in children - with trametinib

Information

Tafinlar
Licence extension / variation
Novartis
Novartis

Development and Regulatory status

Phase II Clinical Trials
Phase II Clinical Trials
Phase II Clinical Trials
Yes
Yes

Category

BRAF inhibitor, given in combination with a MEK inhibitor (trametinib)
In the UK, brain tumours occur in about 5 per 100,000 of children between the ages of 0-9 years. The largest subgroup is astrocytoma, a type of glioma (43% of all brain and CNS tumours in children). Astrocytomas are diagnosed throughout childhood. 76% of astrocytomas are diagnosed as low-grade and 15% as high-grade [1].
BRAF V600 mutation positive low grade glioma (LGG) or high grade glioma (HGG) in children - with trametinib
Oral

Further information

Yes

Evidence based evaluations

Tafinlar BRAF V600E mutation-positive solid tumours, progressed following prior therapy with no other options - with trametinib

Information

Tafinlar
Licence extension / variation
Novartis
Novartis

Development and Regulatory status

None
Phase II Clinical Trials
Launched
Jun 22FDA grants accelerated approval to Tafinlar with Mekinist for any advanced solid tumours with the BRAF V600E mutation. The patient must have progressed following prior therapy and have no alternative treatment options. The pan-tumour indication covers patients as young as 6 years old [1].

Category

BRAF inhibitor, given in combination with a MEK inhibitor (trametinib)
Variable depending on tumour type. BRAF V600E is the most common type of BRAF mutation, making up about 90% of all BRAF-mutant cancers. BRAF mutations have been identified in more than 20 tumour types [1].
BRAF V600E mutation-positive solid tumours, progressed following prior therapy with no other options - with trametinib
Oral

Trial or other data

Jun 22PII ROAR study, which completed in Dec 21, recruited in the US, Canada, Japan, Republic of Korea and Europe (not UK). It included adults with with rare cancers including anaplastic thyroid cancer, biliary tract cancer, gastrointestinal stromal tumor, non-seminomatous germ cell tumour/non-geminomatous germ cell tumour, hairy cell leukaemia, World Health Organisation (WHO) Grade 1 or 2 glioma, WHO Grade 3 or 4 (high-grade) glioma, multiple myeloma, and adenocarcinoma of the small intestine, with BRAF V600E positive-mutations [2].
Jun 22Tafinlar and Mekinist showed tumour shrinkage benefits in several cancer types in two clinical trials. Across 131 patients enrolled in the PII ROAR study and Arm H of the NCI-MATCH trial, the combination triggered responses in biliary tract cancer, high-grade and low-grade glioma, ovarian cancer, small intestine adenocarcinoma and others. Plus, in a small trial (Study X2101), the doublet showed an acceptable safety profile and some tumour shrinkage ability in paediatric patients, mainly those with pre-treated glioma [1].