Alexion Pharmaceuticals, Inc.  announced today that NICE Highly Specialised Technologies Evaluation Committee (EC) has reaffirmed the very significant clinical value of eculizumab (Soliris) for the treatment of atypical hemolytic uremic syndrome (aHUS) and the lack of other effective therapies, and has issued a recommendation that it be nationally commissioned for all patients suffering from this severe and life-threatening genetic disorder.

In the second Evaluation Consultation Document (ECD) released today, the NICE EC again confirmed the 2013 Advisory Group for National Specialised Services (AGNSS) positive assessment as well as the NICE assessment from earlier this year that eculizumab is a very effective treatment for aHUS patients and produces substantial quality-adjusted life year gains of a magnitude rarely seen for any new drug treatment. The Committee recommended the commissioning of eculizumab for aHUS patients subject to the following conditions:

• 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.

“We are pleased that NICE has once again confirmed that patients with aHUS are at constant risk of sudden, progressive and life-threatening damage to vital organs including the kidney and other organs, and that eculizumab is a significant breakthrough for patients with this devastating disorder,” said Leonard Bell, M.D., Chief Executive Officer of Alexion. “Alexion supports the use of eculizumab consistent with the EMA-approved label, which specifically directs that treatment is recommended to continue for the patient’s lifetime, unless the discontinuation of eculizumab is clinically indicated. We believe that it is important that NICE work within its remit and that decisions regarding continuation of eculizumab should be made by the treating physician based on best clinical judgment. We will provide specific comments to NICE to address its conditions and look forward to working to ensure equity such that patients with aHUS in England, like patients with PNH in England, have equal and sustained access to this life-transforming therapy.”

For more details, go to: http://news.alexionpharma.com/press-release/company-news/nice-recommends-commissioning-eculizumab-soliris-all-patients-ahus-englan