Novartis Receives 3 Simultaneous FDA Approvals for Novel Biologic Drug

The FDA recently approved Ilaris (canakinumab) for the treatment of 3 rare types of periodic fever syndromes, making it the only approved biologic treatment for the conditions.

Ilaris is a selective, high-affinity, human monoclonal antibody that inhibits interleukin beta, according to a press release from Novartis. The drug prevents inflammation associated with an over-production of interleukin beta.

It has received simultaneous approval for the treatment of tumor necrosis factor-receptor associated periodic syndrome, hyperimmunoglobulin D syndrome/ mevalonate kinase deficiency, and familial Mediterranean fever, which are all rare autoinflammatory diseases.

The simultaneous approvals are based on findings from the phase 3 CLUSTER clinical trial. Investigators found that Ilaris offers rapid disease control by day 15, and sustained disease control through 16 weeks compared with placebo, according to Novartis.

These results were seen in all patients taking Ilaris with any of the 3 syndromes. Due to the significant results, the FDA granted the drug Breakthrough Therapy status, as well as priority reviews for each of the 3 indications, since the drug addresses an unmet need.

These autoinflammatory disease can cause disabling, persistent fevers that can also cause joint pain, swelling, muscle pain, and skin rashes. A majority of patients show symptoms in early life, but some are diagnosed in adulthood.

Previous treatments included corticosteroids or non-steroidal anti-inflammatory drugs, and were used to manage symptoms. Non-steroidal anti-inflammatory drugs were not able to prevent or change the course of a flare, Novartis reported.

Ilaris has received prior FDA-approval for 2 cryopyrin-associated periodic syndromes (Muckle-Wells synfrome and familial cold autoinflammatory syndrome), and systemic juvenile idiopathic arthritis. The drug has been approved by the European Commission for adult-onset Still’s disease.

Familial Mediterranean fever typically affects people of Eastern Mediterranean ancestry, and affects 1 in 250 to 1 in 1000 individuals in these populations, according to Novartis. Many individuals affected are children.

“Gaining 3 simultaneous FDA approvals of Ilaris is a momentous turning point for people who suffer with these severe, debilitating diseases,” said Paul Hudson, CEO, Novartis Pharmaceuticals. “There is a significant unmet need in these conditions, particularly in children, and Novartis is proud to provide a new treatment option to those who so desperately need it.”