Alexion Says UK Unit Informed AGNSS Recommendation for Soliris Won't Be Followed

Alexion Pharma UK, a subsidiary of Alexion Pharmaceuticals, Inc.

, has been informed by the National Specialised Commissioning Team (NSCT) that the Ministers of Health have decided to not follow a positive recommendation by the Advisory Group for National Specialised Services (AGNSS) when assessing Soliris® (eculizumab) as a treatment for patients with atypical haemolytic uraemic syndrome (aHUS). It is important to underscore that the UK Government developed a specific process and authorised a committee, AGNSS, to evaluate treatments addressing patients with very rare disorders, and that AGNSS concluded that “eculizumab should be routinely nationally commissioned for patients with aHUS.” AGNSS further stated that, “Eculizumab would help save lives and improve the quality of life for children (among whom the condition is particularly prevalent) and adults with atypical haemolytic uraemic syndrome.” However, the Ministers of Health have overruled this expert committee recommendation and instead referred consideration of Soliris for the treatment of patients with aHUS to the National Institute for Health and Clinical Excellence (NICE). aHUS is an unpredictable and life-threatening disease that affects both children and adults. More than 50% of patients with aHUS experience devastating consequences, including kidney failure, require dialysis or die within 1 year of diagnosis.1,2 Soliris was approved by the European Commission in 2011 specifically for the treatment of patients suffering with aHUS and is the only approved treatment option available. The Health Ministers decision to refer the Soliris assessment to NICE comes over one and one-half years after the initial application was submitted by the lead physician expert at Newcastle-upon Tyne Hospitals NHS Foundation Trust, and following the positive recommendation by the authorised committee AGNSS. This action pushes the Health Ministers decision further into the future, and with an indefinite timeframe, despite abundant clinical evidence on the safety and efficacy of Soliris for these patients. Alexion is gravely disappointed by the announcement and the underlying departure by the UK Government from an agreed formal process. The significant delay in the Health Ministers decision directly threatens the lives of aHUS patients in England by causing further and unnecessary delays in access to safe and effective treatment. Alexion is committed to working with the Health Ministers to make Soliris available to children and adults as quickly as possible, and calls upon the Ministers to accept the recommendation of the significant number of experts and lay people called upon to review Soliris without delay, and to reverse his decision and accept the recommendation of AGNSS in the best interests of aHUS patients in England.