First and Only Approved Treatment for atypical HUS, a Chronic and Life-Threatening Ultra-Rare Disease
ONTARIO, March 6, 2013 /CNW/ – Alexion Pharma Canada., a subsidiary of Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN), today announced that Canada’s national healthcare regulatory agency, Health Canada, has approved the use of Soliris® (eculizumab) for the treatment of patients with atypical hemolytic uremic syndrome (atypical HUS) to inhibit complement-mediated thrombotic microangiopathy (TMA). Atypical HUS, or aHUS, is a life-threatening and chronic ultra-rare, genetic disease that progressively damages vital organs, leading to stroke, heart attack, kidney failure and death.1
The morbidity and premature mortality in aHUS is caused by chronic uncontrolled activation of the complement system, resulting in the formation of blood clots in small blood vessels throughout the body, known as TMA.2,3 Despite historical supportive care, more than half of all patients with aHUS die, require kidney dialysis or have permanent kidney damage within 1 year of diagnosis.4 Soliris is the first and only therapy for the treatment of this severe and debilitating condition.
“Soliris has been shown to have a life-changing impact on patients with aHUS and represents a major advance in the treatment of this extremely rare but devastating disease that previously had no treatment options,” said Anne-Laure Lapeyraque, M.D., MSc., Assistant Professor of Paediatrics, Division of Nephrology, at the Hospital Sainte Justine, Université de Montréal. “By directly targeting uncontrolled complement activation, the underlying cause of the progressive organ failure and shortened lifespan for patients with aHUS, Soliris can markedly decrease the TMA process, improve kidney function, and ultimately has the potential to change the course of aHUS.”
“On behalf of the patients and their families who have been waiting for a safe and effective treatment for this life-threatening disorder, we are so pleased that Health Canada approved Soliris in a timely fashion,” said Durhane Wong-Rieger, PhD., President of the Canadian Organization for Rare Disorders. “We hope that the private, provincial and federal drug plans act with equal urgency to make sure patients get access to therapy as soon as possible.”
Health Canada has issued a marketing authorization for adolescents and adults (aged 13-17) with atypical HUS to reduce complement-mediated TMA. Health Canada has also approved Soliris for pediatric patients with atypical HUS under the Notice of Compliance with Conditions (NOC/c) policy based on data collected in a retrospective chart study. As part of this condition, Alexion has agreed to undertake an additional prospective study in pediatric patients, and to provide additional data from all ongoing and future trials of Soliris in atypical HUS.
“This approval means that the provincial governments in Canada now have the opportunity to support patients and families in Canada suffering with aHUS to receive the life-transforming benefits of Soliris,” said John Haslam, General Manager of Alexion Pharma Canada. “We will work with Canada’s public and private healthcare organizations to ensure that children and adults suffering from aHUS have access to Soliris as quickly as possible.”
Soliris was approved by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) in 2011 for the treatment of pediatric and adult patients with aHUS to inhibit complement-mediated TMA. Soliris is also approved in the United States, European Union, Japan and other territories including Canada for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH), a debilitating, ultra-rare and life-threatening blood disorder.
aHUS is a chronic, life-threatening, ultra-rare disease in which a genetic deficiency in one or more complement regulatory genes causes life-long uncontrolled complement activation, resulting in complement-mediated thrombotic microangiopathy (TMA), the formation of blood clots in small blood vessels throughout the body.1,2 Permanent, uncontrolled complement activation in aHUS causes a life-long risk for TMA, which leads to sudden, catastrophic, and life-threatening damage to the kidney, brain, heart, and other vital organs, and premature death.2,3 More than half of all patients with aHUS die, require kidney dialysis or have permanent kidney damage within 1 year of diagnosis.4 Patients with aHUS who receive a kidney transplant commonly experience subsequent systemic TMA, resulting in a 90% transplant failure rate.5
aHUS affects both children and adults. In a large group of aHUS patients, 60% were first diagnosed at younger than 18 years of age.5 Complement-mediated TMA also causes reduction in platelet count (thrombocytopenia) and red blood cell destruction (hemolysis). While mutations have been identified in at least ten different complement regulatory genes, mutations are not identified in 30-50% of patients with a confirmed diagnosis of aHUS.6
Soliris is a first-in-class terminal complement inhibitor developed from the laboratory through regulatory approval and commercialization by Alexion Pharmaceuticals, Inc. Soliris is approved in the US, Canada, European Union, Japan and other countries as the first and only treatment for patients with paroxysmal nocturnal hemoglobinuria (PNH), a debilitating, ultra-rare and life-threatening blood disorder, characterized by complement-mediated hemolysis (destruction of red blood cells).
Soliris is also approved in the US and the European Union as the first and only treatment for patients with atypical Hemolytic Uremic Syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy, a debilitating, ultra-rare and life-threatening genetic disorder characterized by complement-mediated thrombotic microangiopathy (blood clots in small vessels). The effectiveness of Soliris in aHUS is based on the effects on thrombotic microangiopathy (TMA) and renal function. Prospective clinical trials in additional patients are ongoing to confirm the benefit of Soliris in patients with aHUS. Soliris is not indicated for the treatment of patients with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS).
Important Safety Information
The Canadian Product Monograph for Soliris includes a boxed warning: “Soliris increases the risk of meningococcal infections. Meningococcal infections may become rapidly life-threatening or fatal if not recognized and treated early.
- Comply with the most current National Advisory Committee on Immunization (NACI) recommendations for meningococcal vaccination in patients with complement deficiencies,
- All patients must be vaccinated with a meningococcal vaccine at least 2 weeks prior to receiving the first dose of Soliris, unless the risks of delaying Soliris therapy outweigh the risks of developing a meningococcal infection; revaccinate according to current medical guidelines for vaccine use.
- All patients must be monitored for early signs of meningococcal infections, evaluated immediately if infection is suspected, and treated with antibiotics, if necessary.
- Vaccination may not prevent all meningococcal infections.”
Meningococcal infections are the most important adverse reactions experienced by patients receiving Soliris.
The most commonly reported adverse events regardless of causality are headache, nasopharyngitis, hypertension, infections, nausea, diarrhea and arthralgia, each occurring in at least 20 percent of patients.
Please see full Product Monograph for Soliris, including boxed WARNING regarding risk of serious meningococcal infection.
About Alexion Pharmaceuticals, Inc.
Alexion Pharmaceuticals, Inc. is a biopharmaceutical company focused on serving patients with severe and ultra-rare disorders through the innovation, development and commercialization of life-transforming therapeutic products. Alexion is the global leader in complement inhibition and has developed and markets Soliris® (eculizumab) as a treatment for patients with PNH and aHUS, two debilitating, ultra-rare and life-threatening disorders caused by chronic uncontrolled complement activation. Soliris is currently approved in more than 40 countries for the treatment of PNH, and in the United States and the European Union for the treatment of aHUS. Alexion is evaluating other potential indications for Soliris and is developing four other highly innovative biotechnology product candidates.
Safe Harbor Statement
This news release contains forward-looking statements, including statements related to anticipated potential health and medical benefits of Soliris® (eculizumab) for the treatment of patients with aHUS, and the timing of and commercial milestones for Soliris in Canada, and the level and timing of insurance coverage and reimbursement for Soliris in Canada. Forward-looking statements are subject to factors that may cause Alexion’s results and plans to differ from those expected, including for example, decisions of regulatory authorities regarding marketing approval or material limitations on the marketing of Soliris for its current or potential new indications, and a variety of other risks set forth from time to time in Alexion Pharmaceuticals Inc.’s filings with the Securities and Exchange Commission, including but not limited to the risks discussed in Alexion’s Annual Quarterly Report on Form 10-K for the period ended December 31, 2012 and in Alexion’s other filings with the Securities and Exchange Commission. Alexion does not intend to update any of these forward-looking statements to reflect events or circumstances after the date hereof, except when a duty arises under law.
1 Noris M, Remuzzi G: Atypical hemolytic-uremic syndrome. N Engl J Med 2009 361:1676-87.
2 Benz K, Amann K. Thrombotic microangiopathy: new insights. Curr Opin Nephrol Hypertens 2010 May; 19(3):242-7.
3 Tsai HM. The molecular biology of thrombotic microangiopathy. Kidney Int 2006 Jul;70(1):16-23.
4 Caprioli J, Noris M, Brioschi S, et al; for the International Registry of Recurrent and Familial HUS/TTP. Genetics of HUS: the impact of MCP, CFH, and IF mutations on clinical presentation, response to treatment, and outcome. Blood. 2006;108:1267-1279.
5 Bresin E, Daina E, Noris M, et al; International Registry of Recurrent and Familial HUS/TTP. Outcome of renal transplantation in patients with non—Shiga toxin-associated hemolytic uremic syndrome: prognostic significance of genetic background. Clin J Am Soc Nephrol. 2006;1:88-99.
6 Noris M, Caprioli J, Bresin E, et al. Relative role of genetic complement abnormalities in sporadic and familial aHUS and their impact on clinical phenotype. Clin J Am Soc Nephrol. 2010;5:1844-1859.