Exelixis’ once-failed cancer prospect gains FDA nod in kidney cancer

FDA sign

Two years after a failed trial and an uncertain future, Exelixis ($EXEL) has finally turned around its misfortunes after being granted U.S. approval for cabozantinib.

The FDA has now given the drug--named Cabometyx--a license in the second-line setting for patients with advanced renal cell carcinoma (RCC) who have already been given an anti-angiogenic therapy.

The tablet’s approval is based on the Phase III Meteor trial--which met its primary endpoint of improving progression-free survival by nearly double (median PFS of 7.4 months vs. 3.8 months) when compared to Novartis’ ($NSV) Afinitor in second-line RCC patients. The drug also significantly improved the objective response rate compared with its rival.

And in new long-term data posted in February, the drug also met its secondary endpoint of boosting overall survival--the golden standard in oncology trials--against Afinitor, with median OS being  21.4 months in the Cabometyx arm, versus the 16.5 months for those taking the Swiss major’s treatment.

Cabozantinib won approval back in 2012 to treat a rare form of thyroid cancer--the first step in what Exelixis hoped would be a string of regulatory successes that would expand its indications.

But two years later, cabozantinib missed its primary endpoint in a big Phase III trial in prostate cancer, roughly halving Exelixis' market cap and leading the biotech to lay off about 70% of its workforce and shift its bets to kidney cancer.

Things looked bleak for the South San Francisco, CA-based biotech, but this week’s approval brings the drug from treating medullary thyroid cancer--affecting just a few hundred patients a year--to a treatable kidney cancer population of 17,000 in the U.S. and 37,000 around the world.

This is also good news for French drugmaker Ipsen, which last month spent $855 million on Cabometyx--paying $200 million up front for the rights to the drug outside the U.S., Canada and Japan.

The treatment is also in the midst of a Phase III trial in hepatocellular carcinoma with data expected next year--and Ipsen has promised to pay out another $50 million if regulators sign off on that indication. Exelixis is also in line for as much as $545 million in milestone payments plus tiered royalties of up to 26% on all of Ipsen's future Cabometyx sales.

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