Swedish Orphan Biovitrum (STO: SOBI) filed its interleukin-1 (IL-1) receptor antagonist Kineret® (anakinra) for for the indication of neonatal-onset multisystem inflammatory disease (NOMID) with the Federal Drug Administration (FDA) in the US under orphan drug status. The filing is based on a clinical trial sponsored by National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) and NIH Clinical Center with M.D Raphaela T Goldbach-Mansky as the principal investigator of the trial that has been running for almost a decade in US.
Findings reportedly previously suggest the drug might have an effect on hearing loss and perhaps even on mental retardation, just a couple of the effects of the very rare and serious paediatric rheumatologic disease also known as chronic infantile neurologic cutaneous and articular syndrome (CINCA). A majority of the patients diagnosed with the condition belonging to cryopyrin-associated periodic syndromes (CAPS, on which the orphan drug designation was granted in 2010) in the U.S. have participated in the trial.
In other company specific news this week, Biogen Iden NASDAQ:BIIB) and SOBI have initiated two global paediatric clinical trials with their long-lasting recombinant Factor VIII and Factor IX Fc fusion proteins (rFVIIIFc and rFIXFc) in haemophilia A and B. The extended studies are open-label multicenter studies designed to evaluate safety and efficacy of the drugs in previously-treated children under the age of 12 with severe haemophilia A or B. The global registration studies of the compounds in over 12 year old patients are ongoing with data expected during H2 this year.
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