Swedish Orphan Biovitrum (SOBI) announced that the US FDA Office of Orphan Products Development (OOPD) has granted orphan drug designation (ODD) to Kineret for the treatment of cryopyrin-associated periodic syndromes (CAPS).

CAPS is a rare autoinflammatory condition with very severe symptoms and is potentially lethal. CAPS is characterized by excessive production of the endogenous inflammatory protein interleukin 1ß (IL-1ß). Kineret is a recombinant version of the naturally occurring IL-1 receptor antagonist which blocks the IL-1 receptor.

"The orphan drug designation (ODD) is an important step in our efforts to document Kineret for the severe and very debilitating disease CAPS. An ODD gives advantages in FDA assistance, user-fee benefits and, after orphan drug registration, seven years of market exclusivity. This will help us in our efforts to offer this potentially very valuable product to CAPS patients with high unmet medical needs." said Peter Edman, Ph.D., chief scientific officer of Swedish Orphan Biovitrum.

Cryopyrin-associated periodic syndromes are members of a family of autoinflammatory diseases. CAPS is a rare, autosomal dominant disease consisting of three autoinflammatory conditions of varying severity and oftentimes overlapping symptoms. At the milder end it is characterized by life-long, cold-induced inflammatory episodes of fever, rash and malaise. When of intermediate severity, it is typically associated with more intense and enduring flares and morbidity including progressive hearing loss and kidney failure secondary to amyloidosis (a condition where amyloid proteins are abnormally deposited in organs and/or tissues). In the most severe form (NOMID/CINCA) is associated with high mortality and nearly continuous fevers, rash, chronic aseptic meningitis, sensorineural involvement, craniofacial abnormalities, and exuberant bone lesions. The incidence is estimated to be approximately 1:1,000,000 worldwide, but could be underestimated.

CAPS is characterized by excessive or uncontrolled IL-1ß production. IL-1 induces a number of inflammatory responses such as fever, pain sensitization, bone and cartilage destruction and acute plasma protein response. Approximately 20 per cent of untreated children with NOMID/CINCA syndrome die before reaching adulthood.

In December 2008, Swedish Orphan Biovitrum obtained from Amgen the global exclusive rights to Kineret for rheumatoid arthritis as currently indicated in its label. In November 2009, the agreement regarding Swedish Orphan Biovitrum's Kineret license was expanded to include certain orphan indications.

Swedish Orphan Biovitrum is a Swedish based niche specialty pharmaceutical company with an international market presence. The company is focused on providing and developing specialist pharmaceuticals for rare disease patients with high medical needs.