Novartis has presented late-stage data showing that canakinumab, recently rejected by US regulators for gout, shows potential as a treatment for patients with the most serious form of childhood arthritis.
The Swiss major has presented positive results of the first Phase III trial of ACZ885 (canakinumab) in patients with systemic juvenile idiopathic arthritis (SJIA) at the European Pediatric Rheumatology Congress in Bruges. Around 83.7% of patients experienced at least a 30% improvement in symptoms versus 9.8% for placebo and a third of canakinumab patients achieved a 100% improvement compared with 0% for placebo. The four-week study involved 84 patients between the ages of 2 and 19, with active SJIA.
SJIA affects less than one child per 100,000 and is characterised by potentially life-long and recurrent arthritis flares, which can involve skin rash, daily spiking fever, joint pain and swelling. Therapies traditionally used to treat SJIA can only partially mitigate symptoms and do not prevent the long-term damage of the disease, and long-term steroid use designed to treat SJIA can also contribute to slowed growth and delayed puberty.
The results of a second Phase III trial, aimed at determining whether canakinumab can extend the time to next flare and reduce or eliminate corticosteroid use, will be presented later this year. Worldwide regulatory submissions in SJIA are planned for 2012.
ACZ885 is a fully human monoclonal antibody that neutralises interleukin-1 beta, which is a key driver of inflammation in SJIA, Novartis noted. It is already approved (and sold as Ilaris) in Europe, the USA and elsewhere for cryopyrin-associated periodic syndrome (CAPS).
However, this summer, the US Food and Drug Administration emphatically turned down canakinumab as a treatment for gout, amid concerns about serious infections.