Kidney Cancer Drug Approved in U.S. Faces Scrutiny in Europe

Questions are being raised about the effectiveness of axitinib as a life-prolonging second-line treatment for patients with advanced renal cancer.

Manufactured by Pfizer and marketed as Inlyta, Axitinib was approved in the United States in January 2012 and in Europe in September 2012. The U.K.’s National Institute for Health and Clinical Excellence (NICE), which provides guidance to the National Health Service (NHS), recently released a preliminary report recommending against the use of axitinib as a second-line therapy for the treatment of advanced kidney cancer because of how comparisons with other treatments were conducted.

The preliminary decision by the independent Appraisal Committee of NICE was that axitinib should not be recommended for kidney cancer treatment after first-line treatment failure with sunitinib (another kidney cancer drug from Pfizer marketed as Sutent) or a cytokine drug. However, the committee questioned the comparison information: trial data provided by Pfizer included a direct comparison of axitinib to sofafenib, a drug that NICE had previously recommended against.

The trial also failed to persuasively compare axitinib to the “best supportive care”—the current standard of treatment for patients in the United Kingdom using NHS services—because it used simulated and possibly uncertain results from a different study. Because of validity uncertainties, NICE decided not to recommend the drug (for more information about the NICE decision, please visit http://www.nice.org.uk/newsroom/pressreleases/NICEConsultsNewKidneyCancerDrug.jsp.)

The simulated treatment comparison resulted in an estimated median progression-free survival of 4.6 months in the group taking axitinib after first-line treatment with sunitinib compared with best supportive care and 8.3 months for median overall survival in favor of axitinib, but the committee preliminarily rejected the comparison.

“Before we recommend any new treatment we have to be sure the evidence on how well it works is robust and that it is cost effective,” said Andrew Dillon, the NICE’s CEO. “We do not want to divert NHS funds to a treatment that costs more but doesn’t help people live longer.”

In response, Pfizer noted its disappointment with the preliminary guidance, and said that NICE does not currently recommend any targeted therapies for advanced kidney cancer following failure of first-line drugs, just “best supportive care.”

Pfizer’s U.K. head of oncology, Ben Osborn, said that “we believe that in this disease area where there is still a high level of unmet need, axitinib represents good value to the NHS. To this end, we are committed to working through the NICE consultation process to address the uncertainties within this preliminary recommendation.”

Pfizer has high hopes for axitinib worldwide, as it is one of the products that the drug manufacturer is hoping will offset losses. The company’s successful drug Lipitor (atorvastatin) faces competition from generic drugs.

January 2013 (Vol. 5, Number 1)