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
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
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
(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
(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.
(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,
Kim Diamond, 917-699-5093 Director, Corporate Communications
SOURCE: Alexion Pharma Canada Corp
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