Cabozantinib inhibits the activity of tyrosine kinases (including RET, MET and VEGF receptors) involved in cellular function and pathologic processes such as oncogenesis, metastasis, tumour angiogenesis and maintenance of the tumour microenvironment.
The safety and efficacy of cabozantinib were established in a randomised, double-blind trial involving 330 patients with unresectable locally advanced or metastatic medullary thyroid cancer who had radiographic evidence of disease progression within 14 months before study entry.
Patients who received cabozantinib 140mg once daily survived for a median of 11.2 months without tumour progression compared with a median of 4 months in the placebo group (HR 0.28, 95% CI 0.19-0.40, p<0.001). Prior therapy with tyrosine kinase inhibitors had no influence on progression-free survival rates.
The overall response rate in cabozantinib-treated patients was 28%, compared with 0% in those who received placebo (p<0.0001). Estimated responses lasted a median of 14.6 months in the cabozantinib arm (95% CI 11.1-17.5 months).
The most commonly reported serious adverse reactions of cabozantinib were pneumonia, mucosal inflammation, hypocalcaemia, dysphagia, dehydration, pulmonary embolism and hypertension.
Further information: Swedish Orphan Biovitrum