AHUS Canada Applauds Approval of First Treatment for Fatal, Ultra-rare Disease Affecting Children and Adults

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Thursday, March 7, 2013

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 exchange therapies.10

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 devastating consequences."

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.

References

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.

Contact
Beth Daniher
416-924-5700 ext. 4070
beth.daniher@cohnwolfe.ca

Source: AHUS Canada

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