Alexion Pharmaceuticals Inc. publicly announced that the United States’ FDA (Food and Drug Administration) has approved the company’s sBLA (supplemental Biologics License Application) providing regular approval for Soliris® (eculizumab) which is used for the treatment of pediatric and adult patients with aHUS (atypical hemolytic uremic syndrome) to inhibit complement-mediated TMA (thrombotic microangiopathy).

This update reflects Alexion’s fulfillment of post-marketing necessities, including the submission of confirmatory data from two additional prospective clinical trials, including one in children with atypical hemolytic uremic syndrome. The new revised label specifies important long-term clinical benefit which is associated with sustained and chronic Soliris treatment with addition of results with two years of ongoing treatment in aHUS patients. This updated label also comprises data on the use of Soliris treatment before the use of supportive care with either plasma infusion or plasma exchange in prospective clinical trials.

Atypical hemolytic uremic syndrome is a rare disease that affects kidney function. The ultra-rare genetic and progressive disease, aHUS is a defined by lifelong and immediate risk of TMA (thrombotic microangiopathy), which results in vital organ failure and premature death. A first-in-class terminal complements inhibitor, Soliris, specially targets chronic uncontrolled complement activation. It is currently the only and the first approved treatment for adult and children with aHUS in Japan, United States, European Union and other countries.

On 23rd September 2011, FDA had approved Soliris with the Accelerated Approval permission (Subpart E) for this indication. The FDA grants Accelerated Approval to drugs that treat serious conditions which are based on an effect on a surrogate clinical endpoint that is reasonably likely to predict a drug’s clinical benefit. To achieve regular approval from FDA a drug company must generally submit additional data to verify clinical benefit.

Leonard Bell, the Chief Executive Officer of Alexion said that they are pleased that, since 2011, the FDA’s Accelerated Approval process has enabled them to provide Soliris to individuals whose lives have been at risk of the severe clinical manifestations of aHUS and who would otherwise have had no effective or safe treatment options.

He also added that the results which were obtained from the two additional prospective clinical trials further confirmed the benefit, efficacy and safety of chronic Soliris treatment in both children and adults with aHUS.

The latest updated label now includes data that specifically supports the long-term benefit associated with sustained and chronic Soliris treatment. They continue to work with a sense of urgency to bring Soliris to more patients who are suffering from this life-threatening disease worldwide.