Swedish Orphan Biovitrum (Sobi) announced today that the US Food and Drug Administration (FDA) has approved Kineret for the treatment of neonatal-onset multisystem inflammatory disease (NOMID). Kineret is the first and only FDA-approved therapy treatment for NOMID, the most severe form of cryopyrin associated periodic syndromes (CAPS).
CAPS, a life-long and severely debilitating disease, is characterized by an overabundance of an immune system protein, interleukin-1 (IL-1). When left untreated, this disease can cause progressive hearing and vision loss, variable degrees of cognitive impairment and joint contractures.
Kineret, which was approved for NOMID under an Orphan Drug designation, has already been approved for the reduction of signs and symptoms of Rheumatoid Arthritis (RA) in adults since 2001. Through the treatment of Kineret for five years, patients have experienced a reduction in daily symptoms of NOMID, including fever, rash, headache and joint pain and a stabilization in important central nervous system (CNS) functions, such as hearing and vision abilities.
“In order to prevent organ damage that results from untreated disease, we have learned that early diagnosis and the initiation of IL-1 blocking therapy are critical,” said Dr. Raphaela Goldbach-Mansky, the principal investigator of the NOMID study that was conducted at the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), part of the National Institutes of Health (NIH) in Bethesda, Maryland.
Dr. Geoffrey McDonough, the CEO of Sobi said, “The FDA approval of Kineret for NOMID is a significant result from a long-term collaboration with the NIH and patient societies, and marks an important milestone in our effort to bring therapeutics options to patients with rare inflammatory diseases. We look forward to making Kineret more widely available to patients with NOMID in the United States this year.”
About Swedish Orphan Biovitrum (Sobi):
Sobi is an international specialty healthcare company committed to rare diseases. Its mission is to develop and deliver innovative therapies and services to improve the lives of patients. Its product portfolio is primarily focused on inflammation and genetic diseases. Sobi is a pioneer in biotechnology with world-class capabilities in protein biochemistry and biologics manufacturing.