Soliris™ (Eculizumab), the First and Only Proven Therapy for the Treatment of Patient

[Doug Mylie]

Until recently, patients with paroxysmal nocturnal haemoglobinuria (PNH)—an ultra-rare, progressive, and life-threatening blood disorder—had no therapeutic options to address this devastating disease. But now, Soliris™ (eculizumab), a new treatment option for PNH, is available in Canada.

Soliris is the first and only approved medication proven to reduce haemolysis, the underlying cause of the clinical manifestations of PNH. PNH is a progressive and destructive disease that can lead to thrombosis, end organ damage, impaired quality of life—independent of anaemia/transfusion requirements—and mortality.

A genetic mutation in PNH patients leads to the creation of abnormal blood cells that do not have terminal complement inhibitors, making the cells sensitive to destruction by the protein complement. The destruction and loss of these oxygen-carrying red blood cells results in anaemia, as well as fatigue, difficulty in functioning, pain, dark urine, shortness of breath and blood clots.

The safety and efficacy of Soliris in PNH patients has been demonstrated in three multi-national clinical trials: TRIUMPH, SHEPHERD, and E05-001, a long-term extension study. The studies determined that patients with PNH who received Soliris experienced a number of benefits including an immediate and sustained reduction in chronic haemolysis(1,2), fewer blood clots, significant improvements in fatigue levels and overall quality of life(1,2) and a significant reduction in the need for transfusions(2).

In January 2009, Soliris was approved under priority review by Health Canada. Soliris has also been recognized on the international stage for scientific innovation and the positive impact the drug is having on the lives of patients with PNH. Soliris received both the 2009 Prix Galien International Award in the category of medicines for rare diseases and the 2008 Prix Galien USA Award for Best Biotechnology Product.

Any patient who is diagnosed with PNH will have access to Alexion Pharma Canada’s OneSource™ program, a personalized service for patients with PNH and their health care providers. Each patient who enrols in OneSource is assigned a specific OneSource Case Manager who supports the patient immediately and over time, providing a broad range of services from disease education to coordinating care with the physician’s office. Case managers also work with patients and physicians to secure access to Soliris and identify funding options when needed. All case managers are registered nurses who have extensive experience caring for patients.

For more information please contact 1 888 SOLIRIS (888-765-4747) or email Alexion Pharma Canada at AlexionPharmaCanada@alxn.com.
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(1) Brodsky RA, Young NS, Antonioli E, Risitano AM, Schrezenmeier H, Schubert J, et al. Multicenter phase 3 study of the complement inhibitor eculizumab for the treatment of patients with paroxsymal nocturnal hemoglobinuria. Blood 2008 Feb 15;111(4):1840-7.

(2) Hillmen P, Young NS, Schubert J, Brodsky RA, Socie G, Muus P, et al. The complement inhibitor eculizumab in paroxsymal nocturnal heomoglobinuria. N Engl J Med 2006 Sep 21;355(12):1233-43.

[Marrowforums]

Soliris (eculizumab), made by Alexion Pharmaceuticals, Inc., was approved under priority review by Health Canada's Biologics and Genetics Therapeutic Directorate in January. More recently Alexion has said that it has set up a new division to market Soliris in Canada.