- Patients with aHUS and families urge swift access to life-saving drug
TORONTO, March 7, 2013 /CNW/ - Atypical Hemolytic Uremic Syndrome (aHUS)
Canada is thrilled by Health Canada's recent approval of Soliris®
(eculizumab) for the treatment of patients with atypical Hemolytic
Uremic Syndrome (aHUS), 1 a very rare, chronic and life-threatening genetic condition affecting
fewer than 60 patients in Canada.
aHUS leaves a part of the immune system (known as the complement system)
uncontrolled and always active. As a result, the immune system attacks
the body's unhealthy and healthy cells which can cause blood vessel
damage, abnormal blood clotting 2,3 and progressive damage to the body's major organs, leading to heart
attack, stroke, kidney failure and death.4
The management of aHUS has relied on plasma infusion and plasma exchange
therapies with variable results.5 These lifelong therapies are costly, painful and time-consuming, and
have not been studied or approved for the treatment of aHUS.6 If kidney failure has already occurred as a result of aHUS, dialysis is
required, though it is not a curative treatment.7 Within a year of diagnosis, over half of patients will need dialysis,
will have irreversible kidney damage, or will not survive.6 The majority of patients progress to end-stage kidney failure within
three years of diagnosis.8,9
With the approval of Soliris, aHUS patients and their families finally
have a reason for hope.
Sonia DeBortoli knows all too well the destructive force of the
disease. Sonia's 11-year-old son Joshua was diagnosed with aHUS in
March 2012 and experienced kidney failure, internal bleeding and a
blood clot in his groin as a result. He endured several painful hours
of daily dialysis and plasma therapy, and was on prednisone and oxygen.
Then, a chance to join a clinical trial for Soliris restored Joshua's
health so that he no longer needed the other therapies.
"Our whole world changed when Joshua was given Soliris - we now believe
he has a long and healthy future. He is back at school, taking karate
lessons and playing soccer," Sonia says. "We got our little boy back,
he got his life back, and we want the same for anyone who has to deal
with this rare and devastating disease."
A groundbreaking treatment advance for aHUS patients
Soliris (eculizumab) is the first and only pharmaceutical treatment for
aHUS, and is being hailed by experts worldwide as a critical
breakthrough in treating the disease. It has been shown to
significantly improve patients' health and quality of life.10 In clinical trials, Soliris has been proven effective in preventing
blood vessel damage and abnormal blood clotting,11,12 leading to remission and significant improvement in kidney function.5,4 Soliris has also allowed patients to discontinue dialysis and plasma
Soliris is also indicated, and proven safe and effective, for the
treatment of another rare and life-threatening disorder called
paroxysmal nocturnal hemoglobinuria (PNH).13 Canadians living with PNH already have access to Soliris through
private health insurance and provincial drug plans.
Immediate and sustained access to treatment urgently needed
Now that this new treatment option has been approved for the small
number of Canadians living with the devastating symptoms of aHUS,
Soliris must be made immediately accessible to all aHUS patients whose
lives depend on this treatment.
"We are so hopeful that the Common Drug Review will recognize the urgent
need for access to Soliris, and that provincial governments will act
swiftly to provide reimbursement to patients who are in urgent need of
this life-saving treatment," says Tracy MacIntyre, a founder of aHUS
Canada whose daughter is living with aHUS. "Immediate access to the
drug would have a profoundly positive impact on the few Canadians
living with aHUS, while any delay in funding treatment could lead to
About aHUS Canada
aHUS Canada was formed in November 2012 to support Canadian patients and
families living with aHUS. In addition to establishing a Canadian aHUS
community, the group is committed to building public awareness and
understanding of aHUS and advocating for the best possible care and
treatment for patients. For more information, please visit www.ahuscanada.org.
1 Health Canada. Notice of Compliance Information. Accessed on March 5,
2013. Available at: http://webprod5.hc-sc.gc.ca/noc-ac/info.do?no=14168&lang=eng
2 Benz K and Amann K. Thrombotic microangiopathy: new insights. Curr Opin
Nephrol Hypertens. 2010;19(3):242-247.
3 Tsai HM. The molecular biology of thrombotic microangiopathy. Kidney
Int 2006 Jul;70(1):16-23.
4 Noris M and Remuzzi G. Review Article: Atypical Hemolytic-Uremic
Syndrome. N Engl J Med 2009;361:1676-87.
5 Mache C, Acham-Roschitz B, Frémeaux-Bacchi V, et al. Complement
Inhibitor Eculizumab in Atypical Hemolytic Uremic Syndrome. Clin J Am
Soc Nephrol. 2009;4:1312-1316.
6 Caprioli J, Noris M, Brioschi S, et al. Genetics of HUS: the impact of
MCP, CFH, and IF mutations on clinical presentation, response to
treatment, and outcome. Blood. 2006;108:1267-1279.
7 The Kidney Foundation of Canada. Dialysis. Accessed on March 1, 2013.
Available at http://www.kidney.ca/page.aspx?pid=337
8 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.
9 Kavanagh D and Goodship T. Atypical Hemolytic Uremic Syndrome, Genetic
Basis, and Clinical Manifestations. Acquired Hematopoietic Disorders:
Complement-Mediated Blood Disorders. 2011:15-20.
10 Kim J, Waller S and Reid C. Clinical Report: Eculizumab in atypical
haemolytic-uraemic syndrome allows cessation of plasma exchange and
dialysis. Clin Kidney J. 2012;0:1-3.
11 Legendre C, Babu S, Furman R, et al. Safety and Efficacy of Eculizumab
in aHUS Patients Resistant to Plasma Therapy: Interim Analysis from a
Phase 2 Trial. Abstract presented at the 43rd annual meeting of the
American Society of Nephrology, Denver, CO, USA, 16-21 November 2010.
12 Muus P, Legendre C, Douglas K et al. Safety and Efficacy of Eculizumab
in aHUS Patients on Chronic Plasma Therapy: Interim Analysis of a Phase
2 Trial. Abstract presented at the 43rd annual meeting of the American
Society of Nephrology, Denver, CO, USA, 16-21 November 2010.
13 Fremeaux-Bacchi, V. Treatment of atypical uraemic syndrome in the era
of eculizumab. Clin Kidney J (2012) 5: 4-6.
SOURCE: aHUS Canada
For further information:
Cohn & Wolfe
416-924-5700 ext. 4070