The U.S. Food and Drug Administration has approved a drug to help ease the suffering faced by those with certain chronic inflammatory diseases.
Arcalyst (rilonacept, an Interleukin-1 blocker) is now approved for the long term treatment of two Cryopyrin-Associated Periodic Syndromes (CAPS) disorders: Familial Cold Auto-Inflammatory Syndrome (FCAS) and Muckle-Wells Syndrome (MWS).
Symptoms of both of these disorders include inflammation such as joint pain, rash or skin lesions, fever and chills, eye redness or pain, and fatigue in both children and adults; however MWS is associated with more severe inflammation and may include hearing loss or deafness. In addition, some MWS patients may also be affected by the buildup of a protein substance that damages organs and tissue (amyloidosis). The FCAS and MWS disorders affect about 300 people in the United States. CAPS disorders are inherited. Fifty percent of CAPS cases are associated with a gene mutation in the CIAS 1 gene.
"Arcalyst offers new promise for this small patient population suffering disorders associated with Cryopyrin-Associated Periodic Syndromes," said Curt Rosebraugh, M.D., M.P.H., acting director of the FDA's Office of Drug Evaluation II. "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."