Arcalyst: New Orphan Drug FDA-Approved for Treatment of Rare Inflammatory Syndromes

Filed in archive FDA Approvals , News , Treatment on February 28, 2008

The FDA has just approved a new orphan drug for the treatment of rare inflammatory syndromes.




The said dug - Arcalyst - is the first treatment for an extremely rare condition known as Cryopyrin-Associated Periodic Syndrome or CAPS.

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 is a product of Regeneron.