Novartis announced the FDA has granted breakthrough therapy designations for canakinumab to treat three rare periodic fever syndromes — tumor necrosis factor receptor-associated periodic syndrome, hyperimmunoglobulin D syndrome/mevalonate kinase deficiency and familial Mediterranean fever — for patients with an inadequate response to colchicine, according to a company press release.
The decision follows a phase 3 trial with canakinumab (Ilaris, Novartis) in patients with one of the three types of periodic fever syndromes. According to the release, the autoinflammatory conditions can cause joint pain, swelling and rashes and can be life-threatening. Preliminary evidence of efficacy to treat a life-threatening disease is a condition for breakthrough status. Currently, only colchicine is approved for the treatment of familial Mediterranean fever.
“This is an important day for patients, including many children, who are affected by these serious and debilitating syndromes that have no or limited treatment options,” David Epstein, division head at Novartis Pharmaceuticals, said in the release. “Ilaris is a promising medicine under review for these conditions, marking our commitment to making a significant difference to the lives of people with rare diseases.”
According to the release, canakinumab has been approved since 2009 to treat cryopyrin-associated periodic syndrome, Muckle-Wells syndrome and familial cold autoinflammatory syndrome in patients 4 years and older. In 2013, it was approved to treat systemic juvenile idiopathic arthritis in patients 2 years or older.