FDA Greenlights Soliris for the Treatment of Patients with aHus

Alexion Pharmaceuticals said May 5 that the FDA has approved its sBLA for Soliris (eculizumab) for the treatment of adult and pediatric patients with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA).

The update reflects Alexion’s fulfillment of post-marketing requirements, including the submission of confirmatory data from two additional clinical trials, including one in pediatric patients with aHUS, the company said.

aHUS is an ultra-rare, genetic and chronic disease defined by immediate and lifelong risk of TMA, which is a pattern of damage that can occur in the smallest blood vessels, most commonly the kidney and brain. The condition can result in vital organ failure and premature death.

The revised label for Soliris now includes longer-term clinical benefit associated with chronic and sustained treatment with inclusion of results with two years of ongoing treatment in aHUS patients. The new label also includes data on the use of Soliris treatment prior to use of supportive care with either plasma exchange or plasma in prospective clinical trials.

Soliris is the first and only approved treatment for pediatric and adult patients with aHUS.

“The results obtained from the two additional prospective clinical trials further confirm the safety, efficacy and life-transforming benefit of chronic Soliris treatment in both adult and pediatric patients with aHUS,” said Alexion CEO Leonard Bell. “The updated label now includes data that specifically supports the longer-term benefit associated with chronic and sustained Soliris treatment. We continue to work with a sense of urgency to bring Soliris to more patients suffering from this life-threatening disease worldwide.” — Kellen Owings

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