Swiss drugmaker Novartis has launched its biologic Ilaris in the UK for patients with the rare autoimmune autoinflammatory disease CAPS, making it the only available therapy to treat all three forms of the condition.
Cryopyrin-associated periodic syndrome (CAPS) is a life-long disease – the result of a single gene mutation that causes overproduction of interleukin-1 beta – which leads to sustained inflammation and symptoms such as debilitating fatigue, fever, joint pain and conjunctivitis.
Long-term consequences of CAPS include deafness, bone and joint deformities, and central nervous system damage leading to visual loss. In addition, around 25% of patients develop amyloidosis, which can result in renal failure, and 20% of patients with Neonatal-Onset Multisystem Inflammatory Disease (NOMID) – the most severe form of CAPS – die before reaching adulthood.
Novartis’ Ilaris (canankinumab) is a fully human monoclonal antibody that is designed to “switch off” disease activity by targeting and blocking interleukin-1 beta, and so is able to offer long-term control of the illness, according to the firm.
Following its speedy European green light back in October, the drug is now the only treatment approved to treat all three forms of CAPS as well as patients as young as four years old, thereby fulfilling a significant unmet medical need for the estimated 2,500 people in the region who suffer from the disease.
Approval of the drug was based on a solid clinical data package which showed that the agent produced remission of symptoms in up to 97% of CAPS patients, with most of them responding within 24 hours of the first injection. Furthermore, “after two weeks the disease was barely detectable in the blood and the remission of symptoms was sustained for six months,” added Dr Helen J Lachmann, from the UK National Amyloidosis Centre at the Royal Free and University College Medical School, London.
According to Novartis, the launch also signifies “a significant step forward” in its strategy of developing its rare disease portfolio, and while analysts have valued the CAPS market at about $50 million, the group said it believes that the compound also has a lot of potential in a number of other indications.
A spokesperson for the company confirmed to PharmaTimes UK News that a single pack of Ilaris, containing 150mg of canakinumab, costs £9927.80.
