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
"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
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
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
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.
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.
SOURCE: Alexion Pharma Canada Corp
For further information:
Alexion Pharmaceuticals, Inc.:
Irving Adler, 203-271-8210
Executive Director, Corporate Communications
Kim Diamond, 917-699-5093
Director, Corporate Communications