Votubia prescribable for renal angiomyolipoma in TSC

1 February 2013

Votubia (everolimus) is now additionally licensed for the treatment of renal angiomyolipoma in adults with tuberous sclerosis complex (TSC).

Everolimus works via the inhibition of mammalian target of rapamycin, a key serine-threonine kinase known to be upregulated in a number of cancers

Votubia can be prescribed for adult patients who are at risk of complications but do not require immediate surgery.

10mg tablet now available

The recommended dose is 10mg once daily and Novartis has launched a 10mg tablet presentation to support this. The dose should be reduced or treatment interrupted in patients who develop severe or intolerable adverse reactions, including pneumonitis, stomatitis, metabolic events (including hyperglycaemia or dyslipidaemia) or other non-haematological toxicities.

EXIST-2 study

The licence extension is supported by the results of the phase III EXIST-2 study in 118 patients with TSC and at least one angiomyolipoma of ≥3cm diameter confirmed by local radiology assessment. Patients were randomised to receive oral everolimus 10mg once daily or placebo and the primary efficacy endpoint was angiomyolipoma response rate, based on independent central radiological review.

The response rate in the everolimus group was 41.8% compared with 0% in the control group (p<0.0001). Everolimus also produced improvements compared with placebo in both key secondary endpoints measured: time to angiomyolipoma progression and skin lesion response rate.