Regeneron's CAPS drug cleared by FDA

US firm Regeneron Pharmaceuticals says it has received marketing approval from the Food and Drug Administration for Arcalyst (rilonacept) injection for subcutaneous use, an interleukin-1 blocker, for the treatment of cryopyrin-associated periodic syndromes (CAPS), including familial cold auto-inflammatory syndrome (FCAS) and Muckle-Wells syndrome (MWS) in adults and children 12 and older.

The company says that Arcalyst is the only therapy approved for patients with CAPS, a group of rare, inherited, auto-inflammatory conditions characterized by life-long, recurrent symptoms of rash, fever/chills, joint pain, eye redness/pain, and fatigue. Intermittent, disruptive exacerbations or flares can be triggered at any time by exposure to cooling temperatures, stress, exercise, or other unknown stimuli. Regeneron said it expects to launch the drug, previously awarded orphan designation, within the next 30 days.

"Arcalyst offers new promise for this small patient population suffering disorders associated with cryopyrin-associated periodic syndromes," said Curt Rosebraugh, acting director of the FDA's Office of Drug Evaluation II, adding: "the Orphan Drug Act - now in its 25th year - has been tremendously successful in delivering effective treatments to patients with extremely rare, but serious, diseases."