As expected, regulators in the USA have turned down canakinumab, Novartis' new treatment for gout.
The Swiss major has received a complete response letter from the US Food and Drug Administration for ACZ885 (canakinumab) in gouty arthritis patients and has asked for additional information, including clinical data to evaluate the benefit-risk profile in refractory patients. The decision follows a negative vote in June by the agency's Arthritis Advisory Committee.
Then, the panel voted 11-1 against backing ACZ885 to treat gouty arthritis attacks in patients who cannot obtain adequate relief with non-steroidal anti-inflammatory drugs or colchicine, amid concerns about serious infections. It also voted 12-0 against the treatment being used to delay and reduce the frequency of future attacks.
In its response to the CRL, Novartis noted that the FDA panel voted in favour of the overall efficacy of canakinumab, but wants to see additional retreatment data to assess the overall safety profile. ACZ885 is a fully human monoclonal antibody that neutralises interleukin-1 beta and would represent the first new class of therapies in nearly half a century to treat gouty arthritis.
Novartis said it will continue to work with the FDA "on next steps" and the company "remains committed to studying ACZ885 in inflammatory diseases where interleukin-1 beta plays a key role". Novartis submitted the drug in European Union 2010 and in the USA, Canada and Switzerland in the first quarter of 2011.
These submissions were based on trials that showed gouty arthritis patients treated with ACZ885 at the time of an attack experienced superior pain relief at 72 hours and a significant reduction in the risk of new attacks over six months, compared to patients treated with the injectable steroid, triamcinolone acetonide (TA). The drug was generally well tolerated in these studies, with most adverse events being mild to moderate in severity, but side effects including among others infections, were reported more frequently in patients treated with ACZ885 vs those treated with TA.
Canakinumab is already approved (and sold as Ilaris) in Europe, the USA and elsewhere for cryopyrin-associated periodic syndrome (CAPS).