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| |  Eculizumab Approved in Australia for Paroxysmal Nocturnal Haemoglobinuria NEW YORK -- February 25, 2009 -- The Australian Government's Therapeutic Goods Administration has approved the use of eculizumab (Soliris) for the treatment of patients with paroxysmal nocturnal haemoglobinuria (PNH). The safety and efficacy of eculizumab were evaluated in a 26-week, double-blind, placebo-controlled study of 87 patients with PNH who had received at least 4 red blood cell (RBC) transfusions over the past 12 months and had received meningococcal vaccination. The study showed significant improvements in haemoglobin stabilisation and reduction in the number of transfused RBC units, the study's coprimary endpoints (P < .01). Half of the patients treated with eculizumab, but none of the placebo-treated patients achieved haemoglobin stabilisation. The median numbers of transfused RBC units were 10 units for placebo-treated patients and 0 units for eculizumab-treated patients. Eculizumab was generally well tolerated. The most frequent adverse events observed were headache, nasopharyngitis, back pain, and nausea. Patients were also treated with eculizumab in a single-arm, 52-week study and in a long-term extension study. In these longer-term studies, patients showed improvements in measures of haemolysis similar to those observed in the randomised controlled trial. In all eculizumab studies, meningococcal sepsis developed in 2 of the 196 patients receiving the drug. Both had previously received meningococcal vaccination. The Australian product label for eculizumab includes a boxed warning: "[Eculizumab] increases the risk of meningococcal infections. Vaccinate patients with a meningococcal vaccine at least 2 weeks prior to receiving the first dose of [eculizumab]; revaccinate according to current medical guidelines for vaccine use. Monitor patients for early signs of meningococcal infections, evaluate immediately if infection is suspected, and treat with antibiotics if necessary." Prior to beginning eculizumab therapy, all patients and their prescribing physicians are encouraged to enrol in the PNH Registry. The Registry will be an integral part of the monitoring and data collection regarding the safety and effectiveness of eculizumab. SOURCE: Alexion Pharmaceuticals, Inc.
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