Alexion's Soliris® (eculizumab) Receives Marketing Approval in Canada for Patients with atypical HUS

Alexion's Soliris® (eculizumab) Receives Marketing Approval in Canada for 
Patients with atypical HUS 
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. 
About aHUS
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) 
About Soliris
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.

References:

(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.

Alexion Pharmaceuticals, Inc.: Irving Adler, 203-271-8210 Executive Director, 
Corporate Communications

Kim Diamond, 917-699-5093 Director, Corporate Communications

SOURCE: Alexion Pharma Canada Corp

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CO: Alexion Pharma Canada Corp
ST: Ontario
NI: MTC 

-0- Mar/06/2013 11:30 GMT


 
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