Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) and Alexion Pharma International Sàrl (APIS), announced today that the Paul-Ehrlich-Institut (PEI), Germany's healthcare regulatory body for biological products, has authorized initiation of an open-label clinical trial to investigate eculizumab (Soliris®) as a treatment for patients with Shiga-toxin producing E. coli hemolytic uremic syndrome (STEC-HUS), which has resulted from infections by Enterohemorrhagic Escherichia coli (EHEC) in an unusually wide outbreak in Germany that began in May.
EHEC infections are very uncommon, and the STEC-HUS complication of EHEC, which affects only a subset of these patients, is ultra-rare. STEC-HUS, a devastating and life-threatening disease, is caused by uncontrolled complement activation.
As announced on May 30, 2011, in response to requests of German physicians and hospitals who are treating patients with STEC-HUS, Alexion has initiated an eculizumab access program and has been providing eculizumab free of charge throughout the crisis. As the number of cases of STEC-HUS has increased to unprecedented levels, PEI, leading physicians, and Alexion have agreed that implementation of a clinical trial is the best environment to ensure that the investigational therapy is provided to patients in a controlled manner to support safety and potential efficacy in this severe clinical setting. The study aims to include all patients who are receiving eculizumab in the current STEC-HUS outbreak.
"We have an unprecedented number of patients with STEC-HUS who are severely ill with a broad range of clinical manifestations. As we continue to react quickly to this public health crisis, we believe that the best way to evaluate the safety and efficacy of eculizumab in treating STEC-HUS patients is through a controlled study," said Rolf Stahl, M.D., Chairman, Department of Nephrology, University Hospital Hamburg-Eppendorf UKE and lead investigator of the clinical trial. "The purpose of this study is to provide a framework for a coordinated and orderly collection of data to both educate physicians in the current crisis and to learn more about the potential of eculizumab to treat patients with STEC-HUS in the future."
According to the Robert Koch Institute, more than 3,400 people in Germany have been affected by the EHEC outbreak, with over 35 deaths. Approximately 800 confirmed cases of STEC-HUS have been reported in what is likely one of history's worst outbreaks of the disease.
"From the start of the current crisis Alexion moved quickly to respond to physician requests for immediate access to eculizumab. We are now increasing our commitment by working with leading German physicians to implement this clinical trial under the authorization of the Paul-Ehrlich-Institut," said Leonard Bell, M.D., Chief Executive Officer of Alexion. "Alexion is eager to work with the medical community in Germany during this crisis, and to share our two decades of experience in researching and treating complement-based diseases. Our chief objective is to work with German physicians and PEI to learn more about the potential role of terminal complement inhibition with eculizumab as a treatment option for the subset of patients who develop STEC-HUS. Our experience with ultra-rare disorders is especially relevant here considering the extremely small numbers of patients who will ever develop STEC-HUS on a global basis."
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