Today, the National Institute for Health and Care Excellence (NICE) have recommended cabozantinib (Cabometyx) as a new first-line treatment option for people with intermediate- or poor-risk advanced renal cell carcinoma (RCC) for use within NHS England and Wales.

Cabozantinib is a once-daily oral tablet which targets multiple pathways involved in the growth of tumours. Cabozantinib has been shown to significantly extend progression free survival compared with sunitinib, when given to untreated advanced RCC patients. Because of its multi-targeted approach, cabozantinib would likely have additional benefits for some groups of patients.

First-line cabozantinib for advanced RCC was originally rejected by NICE in June this year and today’s recommendation is a reversal of that earlier decision.

The approval is based on data from the phase 2 CABOSUN trial, which showed improvements in progression-free survival (PFS) for patients on cabozantinib compared to those who were given sunitinib (Sutent).

In the CABOSUN study, first-line treatment with cabozantinib reduced the risk of progression or death by 52% compared with sunitinib for patients with advanced RCC. The median PFS was 8.6 months with cabozantinib versus 5.3 months for sunitinib.

Although not discussed during the appraisal meetings, there is anecdotal evidence that cabozantinib is particularly effective against bone metastasesKidney Cancer Support Network (KCSN) has heard from a number of patients who confirm this activity, and clinicians are also recommending cabozantinib specifically for patients with bone metastases. The recommendation of cabozantinib for use within NHS England and Wales is, therefore, fantastic news for this group of patients, and is a welcome addition to the arsenal of drugs now available to clinicians for the first-line treatment of advanced kidney cancer.

Recommendation of cabozantinib for use as a first-line treatment of advanced kidney cancer increases the options available to clinicians and patients for managing this disease. Current first-line treatments offer an important, but sometimes short-lived period of stability, but not all patients respond to these treatments and most patients become refractory after a period of time. Biomarkers for the treatment of RCC are yet to be identified, and unfortunately clinicians are not able to predict which patients will respond to which drug. Therefore, a choice of treatment alternatives in the first-line will enable patients to continue managing their disease, and to maintain quality of life.

Cabozantinib will be available through NHS England within 3 months of the date of publication of this decision, and through NHS Wales within 2 months of the publication date.

Read the final appraisal document on the NICE website here

Read the article in PMLive here

Read article in PharmaTimes here