Sobi (STO: SOBI) today announced that the US Food and Drug Administration (FDA)
has approved Kineret® (anakinra) for the treatment of children and adults with
neonatal-onset multisystem inflammatory disease (NOMID). Kineret® is the first
and only FDA-approved therapy for NOMID, the most severe form of cryopyrin
associated periodic syndromes (CAPS).
This is the first approval allowing the use of Kineret in children. Kineret was
approved for NOMID under an Orphan Drug designation. A priority review was
granted by the FDA based on the product's potential to provide a significant
advance in therapy for the NOMID patient population where no adequate therapy
exists. Sobi will provide a prefilled syringe with a graduated label to allow
flexible dosing in children. Kineret has been approved for the reduction of
signs and symptoms of Rheumatoid Arthritis (RA) in adults since 2001.
CAPS is, in its most severe form of NOMID, a life-long and severely debilitating
disease. The disease is associated with an overproduction of an immune system
protein known as interleukin-1 (IL-1). Untreated patients develop progressive
hearing and vision loss, variable degrees of cognitive impairment and joint
contractures. Treatment of NOMID patients for 5 years with Kineret demonstrated
that, in addition to controlling the daily symptoms of the disease such as
fever, rash, headache and joint pain, important central nervous system (CNS)
functions such as hearing and vision remain stable and do not progress on
treatment. "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, MD, USA.
Dr. Geoffrey McDonough, 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 CAPS and NOMID
Cryopyrin associated periodic syndromes (CAPS) are a group of rare inherited
autoinflammatory diseases caused by autosomal dominant mutations in a gene
called NLRP3. CAPS is characterized by uncontrolled overproduction of IL-1beta.
IL-1 induces a number of inflammatory responses such as fever, pain
sensitization, bone and cartilage destruction and acute plasma protein
responses. In the most severe form NOMID, also called chronic infantile
neurologic cutaneous and arthritis syndrome (CINCA) in Europe, it is associated
with increased mortality and fever, rash, chronic aseptic meningitis,
sensorineural hearing loss, craniofacial abnormalities, and bone lesions. When
of intermediate severity, the disease is typically associated with episodic,
intense and enduring flares and morbidity, including progressive hearing loss
and kidney failure secondary to amyloidosis (a condition where amyloid proteins
are deposited in organs and/or tissues). The mildest form presents with cold-
induced episodes of fever, rash and malaise. The incidence of CAPS is estimated
to be 1:1,000,000 worldwide.
About Kineret (anakinra)
Kineret is a recombinant protein drug approved for the treatment of children and
adults with NOMID, and the reduction in signs and symptoms and slowing the
progression of structural damage in moderately to severely active rheumatoid
arthritis (RA), in patients 18 years of age or older who have failed one or more
disease modifying antirheumatic drugs (DMARDs). Kineret blocks the biological
activity of IL-1 by binding to the interleukin-1 type 1 receptor, expressed in a
wide variety of tissues and organs. IL-1 is a key mediator of inflammation and
driver of autoinflammatory diseases in both adults and children. For more
information on Kineret see the Prescribing Information. (www.kineretrx.com)
For further information, please contact:
Birgitte Volck, Senior Vice President Development, Chief Medical Officer Tel:
+46 8 697 20 94
About Swedish Orphan Biovitrum (Sobi)
Sobi is an international specialty healthcare company dedicated to rare
diseases. Our mission is to develop and deliver innovative therapies and
services to improve the lives of patients. The product portfolio is primarily
focused on inflammation and genetic diseases, with three late stage biological
development projects within hemophilia and neonatology. We also market more than
40 specialty and rare disease products for partner companies. Sobi is a pioneer
in biotechnology with world-class capabilities in protein biochemistry and
biologics manufacturing. In 2011, Sobi had total revenues of SEK 1.9 billion (€
214 M) and about 500 employees. The share (STO: SOBI) is listed on NASDAQ OMX
Stockholm. More information is available at www.sobi.com.
Sobi Press Release on January 8, 2013, in PDF format:
http://hugin.info/134557/R/1668782/541947.pdf
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Source: Swedish Orphan Biovitrum AB (publ) via Thomson Reuters ONE
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