WASHINGTON (dpa-AFX) - Alexion Pharmaceuticals Inc. (ALXN) announced that the National Institute for Health and Care Excellence or NICE Highly Specialised Technologies Evaluation Committee or EC has recommended that Soliris (eculizumab) be commissioned for all patients in England suffering from atypical hemolytic uremic syndrome or aHUS, a severe and life-threatening ultra-rare disorder.
In its final evaluation determination, NICE has recommended Soliris for use within the National Health Service or NHS in England as the first and only treatment for patients with aHUS.
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 November 2011 by the European Commission for the treatment of patients with aHUS. The Clinical Particulars section of the EU label states that 'Soliris treatment is recommended to continue for the patient's lifetime, unless the discontinuation of Soliris is clinically indicated,' as described in the Special warnings and precautions for use subsection.
As noted by NICE in its final evaluation determination, after stopping Soliris, severe TMA complications were reported including graft failure needing hemodialysis, renal insufficiency, end-stage renal failure and respiratory distress needing intubation.
NICE recommended that the certain 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.
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 or TMA, the formation of blood clots in small blood vessels throughout the body. 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.
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