Strong results for Novartis drug in rare autoimmune disease
pharmafile | October 30, 2008 | News story | Research and Development, Sales and Marketing |Â Â NovartisÂ
Novartis' development drug ACZ885 (canakinumab) has turned in impressive results in trials for rare medical conditions.
It showed sustained remission in children and adults suffering from potentially life-threatening auto inflammatory diseases called Cryopyrin-Associated Periodic Syndromes (CAPS).
And in Systemic Juvenile Idiopathic Arthritis (SIJA), the most severe form of arthritis in children, ACZ885 achieved substantial clinical improvement within 15 days in a phase I/II study.
ACZ885 is a fully human monoclonal antibody which blocks interleukin-1 beta (IL-1 beta), the form of the interleukin-1 protein that sustains auto inflammatory diseases such as CAPS and its thought to play a pivotal role in SJIA.
Orphan drug status has been granted to ACZ885 in the European Union and US for treating CAPS and SJIA.
In the CAPS trial, after an initial single dose by subcutaneous injection patients went through to a randomised six-month, double-blind, placebo-controlled study.
No patients in the ACZ885 group experienced a further flare-up of the disease compared with 81% in the placebo group.
The treatment was needed only every two months to maintain the response.
Symptoms of CAPS include fever, skin rash, painful joints and severe headache but there are also more debilitating complications like hearing loss and amyloidosis.
The latter can lead to kidney failure requiring dialysis or transplant.
The manufacturer hopes ACZ885 will have a broad application: in the SJIA trial children aged four to 19 years old were treated, with four of them achieving complete remission.
The disease can cause joint and bone disability and grave complications such as Macrophage Activated Syndrome.
Novartis says studies of ACZ885 are also currently under way in a more common inflammatory disease, rheumatoid arthritis (RA), using biomarkers in an attempt to personalise treatment.
The data was presented at the American College of Rheumatology meeting in San Francisco.
"Current treatments are not always effective and are also short-acting and often poorly tolerated by patients," said Professor Philip Hawkins of the National Amyloidosis Centre at the Royal Free and University College Medical School, London. "The rapid and long-lasting remission induced by ACZ885 is an important and much-needed development for both children and adults with CAPS."
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