FDA approves Novartis' Ilaris for rare disease

The FDA has approved Novartis' Ilaris (also known as canakinumab or ACZ885), a treatment for children and adults with cryopyrin-associated periodic syndrome (CAPS). CAPS is a rare auto-inflammatory disorder caused by a single gene mutation that leads to overproduction of interleukin-1 beta (IL-1ß). Long-term consequences of the disease may be serious and potentially fatal, and there are limited treatment options for patients who have the disorder. Only 300 cases of CAPS have been diagnosed in the U.S, though there are potentially many more undiagnosed patients with the condition due to poor disease recognition.

Ilaris--a monoclonal antibody--works by blocking IL-1ß. Data from a Phase III study demonstrated remission in more than 90 percent of Ilaris-treated patients with cryopyrin-associated periodic syndrome. The dosing schedule is once every eight weeks, which is less often than the current approved therapy.

"Until now, treatments for CAPS patients have been limited to traditional inflammatory-disease medications that work by suppressing the entire immune system, and newer therapies that control the disease better but require more frequent injections," said Hal Hoffman, MD, Associate Professor of Pediatrics and Medicine at University of California. "With rapid and sustained response, good tolerability, and a significantly less-frequent dosing schedule, Ilaris represents an important treatment advance for children and adults with CAPS."

- here's Novartis' release

Suggested Articles

All 12 members of an FDA advisory committee voted to recommend the approval of teprotumumab for a rare, autoimmune eye disease.

Early data out of former Fierce 15 winner Gritstone Oncology have been heralded as a big win for the early-stage biotech by analysts.

Biogen will drop work on gosuranemab in progressive supranuclear palsy but continue on in Alzheimer's.