FDA grants supplemental approval for Kineret to treat deficiency of IL-1 receptor antagonist. Swedish Orphan Biovitrium.

Sobi, an international biopharmaceutical company dedicated to rare diseases, announced that the FDA has approved the supplemental Biologics License Application (sBLA) for Kineret(anakinra) for the treatment of deficiency of IL-1 receptor antagonist (DIRA). Kineret was originally approved by FDA in November 2001 for moderate to severe active rheumatoid arthritis and in January 2013 for a severe form of Cryopyrin-Associated Periodic Syndromes (CAPS), known as NOMID. DIRA ( Deficiency of IL-1 receptor antagonist) is an ultra-rare, autoinflammatory disease caused by a genetic mutation in the IL1RN gene, which encodes the interleukin-1 receptor antagonist (IL-1Ra) protein. In patients with DIRA, the deficiency of IL-1Ra leads to unopposed action of IL-1 signaling, resulting in life-threatening systemic inflammation with skin and bone involvement.