Novartis gets three FDA approvals for expanded use of biologic drug to treat rare autoinflammatory diseases

Novartis announced today that the US Food and Drug Administration (FDA) has granted three simultaneous approvals for the expanded use of Ilaris^® (canakinumab) to treat three rare and distinct types of Periodic Fever Syndromes.

Ilaris is the first and only FDA-approved biologic treatment for patients with Tumor Necrosis Factor Receptor-Associated Periodic Syndrome (TRAPS), Hyperimmunoglobulin D Syndrome (HIDS)/Mevalonate Kinase Deficiency (MKD) and Familial Mediterranean Fever (FMF). All three conditions are part of a group of rare autoinflammatory diseases called Periodic Fever Syndromes, which are also referred to as Hereditary Periodic Fevers (HPF). The most common syndrome is FMF, which mainly affects people of Eastern Mediterranean ancestry. It affects 1 in 250 to 1 in 1,000 individuals in these populations, many of whom are children.

"We are grateful to the scientists, clinical trial investigators and all associates who worked tirelessly in support of patients to gain three simultaneous FDA approvals of ILARIS," said Fabrice Chouraqui, President of Novartis Pharmaceuticals Corporation, "It is through their hard work that Novartis can offer a desperately needed new treatment option to the adults and children who suffer from these debilitating conditions and continue to serve the rare disease community."

The FDA approvals are based on results from the pivotal Phase III CLUSTER study which showed rapid (at Day 15) and sustained disease control with Ilaris compared to placebo through 16 weeks, in patients with either TRAPS, HIDS/MKD or FMF. As a result of the positive data, the FDA granted Ilaris Breakthrough Therapy status and priority reviews for each of the three Periodic Fever Syndrome conditions.

Periodic Fever Syndromes are a group of rare autoinflammatory diseases that cause disabling and persistent fevers which may be accompanied by joint pain, swelling, muscle pain and skin rashes with complications that can be life-threatening.

"Adults and children living with TRAPS, HIDS/MKD or FMF often experience extensive delays in diagnosis because the disorders are so rare that many physicians are unaware of them," said Hal M. Hoffman, M.D., chief of Pediatric Allergy, Immunology, and Rheumatology at Rady Children's Hospital-San Diego and University of California San Diego. "Following diagnosis, our goal is to get patients treated as soon as possible and that has been challenging due to the lack of available treatment options. That's what makes these three approvals for ILARIS so important for patients."

Ilaris is already approved and marketed in the US as an effective and well-tolerated treatment for another Periodic Fever Syndrome condition – Cryopyrin-Associated Periodic Syndromes (CAPS), and another autoinflammatory condition – Systemic Juvenile Idiopathic Arthritis (SJIA).