EU regulators back Ipsen cancer drug in new liver cancer use

Ipsen’s bid to build a cancer franchise around its Cabometyx (cabozantinib) is gathering pace after European regulators backed it in a new indication of second line liver cancer – a use where BMS rival immunotherapy drug Opdivo has failed to gain traction.

The European Commission is almost certain to expand the drug’s label following positive opinion from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) scientific committee this week.

BMS’ cancer immunotherapy Opdivo has not been approved in Europe in second line hepatocellular carcinoma (HCC) in patients already treated with Bayer’s Nexavar (sorafenib), after the manufacturer withdrew its filing last year.

The decision means a new competitor for Bayer’s Stivarga (regorafenib), which was approved in Europe in the second line liver cancer indication last year.

Already approved in two indications in second line advanced kidney cancer, Ipsen charges £5,143 for a 30-day supply of the daily pill, although the manufacturer provides the drug at a confidential discount to the NHS under a private agreement.

Sales of Cabometyx are mounting after approval in advanced renal cell carcinoma, after treatment with Bayer’s Nexavar.

In its half-year results the drug generated revenues of 62 million euros (£55 million), and with the new liver cancer indication in Europe this looks likely to grow.

Developed in partnership with Exelixis, Ipsen has rights to Cabometyx outside of the US and Japan.

European approval will also trigger a $40 million milestone payment to Exelixis, which is entitled to $545 million in potential commercial milestone payments and tiered royalties of up to 26% on Ipsen’s net sales of the drug.

There could be more to come from Cabometyx, which is in early stage trials in solid tumours in combination with Roche’s immunotherapy Tecentriq, and in late-stage trials in first-line renal cell carcinoma with Opdivo.

Alexandre Lebeaut, chief scientific officer at Ipsen, said: “The global burden of liver cancer is increasing and despite the recent introduction of new agents, it remains the second leading cause of cancer mortality worldwide.

“Following today’s positive CHMP opinion and if approved by the European Commission, Cabometyx as monotherapy will give patients with HCC a much-needed new oral therapeutic option.”

The EMA filing is based on the results of the global placebo-controlled phase III CELESTIAL trial which met its primary endpoint of overall survival (OS), with Cabometyx providing a statistically significant and clinically meaningful improvement in OS compared with placebo in patients with advanced hepatocellular carcinoma (HCC) who have been previously treated with Nexavar.