- Final Evaluation Determination Confirms that Soliris is a "Very Effective Treatment for aHUS with Significant Value to Patients" -
- Final Evaluation Determination Includes the Same Conditions Discussed in Draft Recommendation, Including Establishment of an Expert Center and a Robust System to Monitor Patients Receiving Treatment -
"Today's decision is an important victory for patients with aHUS and physicians who now have assurance that they will have access to the life-transforming efficacy of Soliris. We are pleased that Soliris will be made available on the NHS for patients with aHUS and we commend NHS
In the final evaluation determination published today, the NICE EC again confirmed that Soliris represents an important treatment option of significant value to patients with aHUS. The Committee recommended the commissioning of Soliris, within its marketing authorization, for aHUS subject to the conditions provided in the evaluation consultation document released in September. Soliris was approved in
"Patients with aHUS are at constant risk of sudden, progressive and life-threatening damage to vital organs including the kidney and other organs," said
NICE recommended that the following arrangements be in place as conditions for the funding of Soliris for patients with aHUS:
- Coordination of the use of eculizumab through an expert center;
- Monitoring systems to record the number of people with a diagnosis of atypical hemolytic uremic syndrome, the number of people who receive eculizumab, and the dose and duration of treatment for these people;
- A national protocol for starting and stopping eculizumab for clinical reasons;
- And a research programme with robust methods to evaluate when stopping treatment or dose adjustment might occur.
About aHUS
aHUS is a chronic, ultra-rare, and life-threatening disease in which a life-long and permanent genetic deficiency in one or more complement regulatory genes causes chronic uncontrolled complement activation, resulting in complement-mediated thrombotic microangiopathy (TMA), the formation of blood clots in small blood vessels throughout the body.2,3 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.2,4 Seventy-nine percent of all patients with aHUS die, require kidney dialysis or have permanent kidney damage within three years after diagnosis despite plasma exchange or plasma infusion (PE/PI).5 Moreover, 33 to 40 percent of patients die or progress to end-stage renal disease with the first clinical manifestation of aHUS despite PE/PI.5,6 The majority of patients with aHUS who receive a kidney transplant commonly experience subsequent systemic TMA, resulting in a 90 percent transplant failure rate in these TMA patients.7
aHUS affects both children and adults. 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 percent of patients with a confirmed diagnosis of aHUS.5
About Soliris®
Soliris is a first-in-class terminal complement inhibitor developed from the laboratory through regulatory approval and commercialization by Alexion. Soliris is approved in the U.S. (2007),
More information including the full U.S. prescribing information on Soliris is available at www.soliris.net.
Important Safety Information
The U.S. product label for Soliris includes a boxed warning: "Life-threatening and fatal meningococcal infections have occurred in patients treated with Soliris. Meningococcal infection may become rapidly life-threatening or fatal if not recognized and treated early [see Warnings and Precautions (5.1)]. Comply with the most current
In patients with PNH, the most frequently reported adverse events observed with Soliris treatment in clinical studies were headache, nasopharyngitis (runny nose), back pain and nausea. Soliris treatment of patients with PNH should not alter anticoagulant management because the effect of withdrawal of anticoagulant therapy during Soliris treatment has not been established. In patients with aHUS, the most frequently reported adverse events observed with Soliris treatment in clinical studies were headache, diarrhea, hypertension, upper respiratory infection, abdominal pain, vomiting, nasopharyngitis, anemia, cough, peripheral edema, nausea, urinary tract infections, and pyrexia. Soliris is not indicated for the treatment of patients with Shiga-toxin E. coli-related hemolytic uremic syndrome (STEC-HUS). Please see full prescribing information for Soliris, including BOXED WARNING regarding risk of serious meningococcal infection.
About Alexion
Alexion is a biopharmaceutical company focused on serving patients with severe and 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 nearly 50 countries for the treatment of PNH and in nearly 40 countries for the treatment of aHUS. Alexion is evaluating other potential indications for Soliris in additional severe and ultra-rare disorders beyond PNH and aHUS, and is developing other highly innovative biotechnology product candidates, including asfotase alfa, across multiple therapeutic areas. This press release and further information about Alexion can be found at: www.alexionpharma.com.
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Safe Harbor Statement
This news release contains forward-looking statements, including statements related to potential health and medical benefits of Soliris® (eculizumab) for the treatment of patients with aHUS and PNH, pricing for Soliris in
References:
1 Soliris eMPC. Available at http://www.medicines.org.uk/emc/medicine/19966. Last downloaded on
2 Benz K, Amann K. Thrombotic microangiopathy: new insights. Curr Opin Nephrol Hypertens. 2010;19(3):242-7
3 Ariceta G, Besbas N, Johnson S, et al. Guideline for the investigation and initial therapy of diarrhea-negative hemolytic uremic syndrome. Pediatr Nephrol. 2009;24:687-96.
4 Tsai HM. The molecular biology of thrombotic microangiopathy. Kidney Int. 2006;70(1):16-23
5 Noris M, Remuzzi G. Atypical hemolytic-uremic syndrome. N Engl J Med. 2009;361:1676-87.
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;5:1844-59
7 Bresin E, Daina E, Noris M, et al. Outcome of renal transplantation in patients with non-Shiga toxin-associated hemolytic uremic syndrome: prognostic significance of genetic background.
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