Soliris (Eculizumab) granted orphan drug designation in Europe and U.S. for atypical hemolytic uremic syndrome

Alexion Pharmaceuticals, Inc. has announced today that Soliris® (eculizumab), its first-in-class complement inhibitor, has been granted Orphan Medicinal Product Designation by the European Commission for the treatment of patients with atypical Hemolytic Uremic Syndrome (aHUS). AHUS is an ultra-rare, inherited, and life-threatening complement-inhibitor deficiency disease that often progresses to end-stage kidney disease or failure. The U.S. Food and Drug Administration granted orphan drug designation to Soliris for the same indication in May 2009. Soliris is not approved for the treatment of patients with aHUS.

Alexion is currently enrolling patients at the initial sites in four clinical studies of Soliris as an investigational treatment for adolescent and adult patients with aHUS. Clinical studies are also currently being planned to investigate the use of Soliris as a treatment for children with aHUS. If Soliris is approved for the treatment of patients with aHUS in Europe or the U.S., orphan-drug status would entitle Alexion to 10 years of market exclusivity in Europe and seven years of market exclusivity in the U.S. for this use of Soliris.

Soliris is approved in the United States, European Union, Australia and Canada as a treatment for patients with paroxysmal nocturnal hemoglobinuria (PNH), an ultra-rare, debilitating, and life-threatening blood disorder. Soliris has also been designated as an orphan drug in these countries, as well as in Japan, for the treatment of patients with PNH.

"These additional orphan drug designations for Soliris underscore the unmet need faced by patients living with aHUS, an ultra-rare, genetic and life-threatening disease that destroys patients’ kidneys,” said Leonard Bell, M.D., Chief Executive Officer of Alexion. “In response to the work of researchers and inquiries from practicing physicians, we are increasingly focusing our resources to investigate Soliris as a treatment for patients with aHUS.”

About the aHUS Clinical Studies

Atypical Hemolytic Uremic Syndrome is characterized by chronic inflammation, hemolysis (red blood cell destruction), thrombocytopenia (reduced circulating platelets), and microangiopathy (damage in small blood vessels), particularly in the kidney and brain, often progressing to end-stage kidney disease or failure. Like PNH, aHUS is caused by a deficiency in normally occurring complement inhibitor proteins. Typically, patients with aHUS have genetic mutations in one of several complement inhibitor proteins that lead to uncontrolled complement activation. Excessive complement activation may contribute to severe inflammation of the blood vessels and blood clotting through the activation of white blood cells, platelets, and the endothelial cell lining of blood vessels. (1)

The prognosis for patients with aHUS is generally poor. Approximately 70 percent of patients with the most common mutation experience chronic renal insufficiency, chronic dialysis, or death within one year of the first clinical episode. (2) Despite current best supportive care, following kidney transplantation, recurrent aHUS causes kidney transplant failure in up to approximately 60 to 90 percent of patients. (3)

Alexion is currently enrolling patients at the initial sites in four prospective, open-label clinical studies of eculizumab as a treatment for patients with aHUS in North America and multiple European countries: two studies of patients who are plasma therapy sensitive (one in adults and one in adolescents) and two studies of patients who are plasma therapy resistant (one in adults and one in adolescents). In addition to the ongoing trials, clinical studies are currently being planned to investigate the use of eculizumab as a treatment for children with aHUS. Physicians, patients and care givers who are interested in participating in these clinical trials can learn more by contacting Alexion by e-mail at [email protected], or by visiting the Alexion website at and clicking on the clinical trials link. The ongoing trials are also posted to the website maintained by the U.S. National Institutes of Health.

Important Safety Information

Soliris is generally well tolerated. The most frequent adverse events observed in clinical studies were headache, nasopharyngitis (a runny nose), back pain and nausea. Treatment with Soliris should not alter anticoagulant management because the effect of withdrawal of anticoagulant therapy during Soliris treatment has not been established.

The U.S. product label for Soliris also includes a boxed warning: "Soliris increases the risk of meningococcal infections. Meningococcal infection may become rapidly life-threatening or fatal if not recognized and treated early. Vaccinate patients with a meningococcal vaccine at least two weeks prior to receiving the first dose of Soliris; 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." During clinical studies, two out of 196 vaccinated PNH patients treated with Soliris experienced a serious meningococcal infection. Prior to beginning Soliris therapy, all patients and their prescribing physicians are encouraged to enroll in the PNH Registry, which is part of a special risk-management program that involves initial and continuing education and long-term monitoring for detection of new safety findings.

(1) Ståhl A, Vaziri-Sani F, Heinen S, Kristoffersson A-C, Gydell K-H, Raafat R, Gutierrez A, Beringer O, Zipfel PF, and Karpman D. Factor H dysfunction in patients with atypical hemolytic uremic syndrome contributes to complement deposition on platelets and their activation. Blood. 2008;111:5307-5315.

(2) Caprioli J, Noris M, Brioschi S, Pianetti G, Castelletti F, Bettinaglio P, et al. Genetics of HUS: the impact of MCP, CFH, and IF mutations on clinical presentation, response to treatment, and outcome. Blood. 2006 Aug 15;108(4):1267-79).

(3) Loirat C, Noris M, Fremeaux-Bacchi V. Complement and the atypical hemolytic uremic syndrome in children. Pediatr Nephrol. 2008 Nov;23(11):1957-72.


The opinions expressed here are the views of the writer and do not necessarily reflect the views and opinions of News Medical.
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