Biologic therapeutic product maker Alexion Pharmaceuticals, Inc. (ALXN) said Friday that the U.S. Food and Drug Administration has approved its drug Soliris for the treatment of all adults and children with atypical hemolytic uremic syndrome or aHUS, a rare and chronic blood disease that can lead to kidney failure and is also associated with increased risk of death and stroke.
The company noted that Soliris, generically known as eculizumab, is the first and only approved treatment for children and adults suffering with atypical hemolytic uremic syndrome or aHUS.
aHUS is a chronic, ultra-rare, life-threatening disease characterized by thrombotic microangiopathy or TMA, the formation of blood clots in small blood vessels throughout the body. It can cause a reduction in platelet count and also cause life-threatening damage to the kidney, brain, and other vital organs that can lead to stroke, heart attack, kidney failure and death.
Despite current supportive care, more than half of all patients with aHUS die, require kidney dialysis or have permanent kidney damage within 1 year of diagnosis.
Larry Greenbaum, Director of Pediatric Nephrology at Emory University and Children's Healthcare of Atlanta said, "Soliris directly targets uncontrolled complement activation, the underlying cause of the progressive organ failure and shortened life span of patients with aHUS, an extremely rare and devastating disease. The FDA approval of Soliris in aHUS marks the most important advance that has been made for patients and families with this disease."
The FDA approval of Soliris in aHUS is based on data from two prospective pivotal Phase 2 open-label clinical trials in adolescent and adult patients with aHUS, and a third retrospective study in children, adolescents, and adults with aHUS.
In early June, the FDA had granted Alexion's request for priority review of its supplemental Biologics License Application, or sBLA, for Soliris as a treatment for patients with aHUS. The FDA's priority review program provides for an expedited six-month review of drugs that may offer major advances in treatment or that provide a treatment when no adequate therapy exists.
The FDA said that the therapy also is being approved under its accelerated approval program, designed to provide patients with earlier access to promising new drugs followed by further studies to confirm the drug's clinical benefit.
Earlier today, Alexion said that a Committee for Medicinal Products for Human Use or CHMP of the European Medicines Agency has adopted a positive opinion for the extension of use of Soliris to include the treatment of pediatric and adult patients with aHUS in Europe.
Alexion's Soliris, a first-in-class terminal complement inhibitor, is approved in both the U.S. and the European Union in 2007, and Japan in 2010, as the only drug specifically indicated for the treatment of patients with paroxysmal nocturnal hemoglobinuria, or PNH, a debilitating, life-threatening, and ultra-rare blood disorder.
Soliris is classified as an orphan drug. Orphan drugs are those that demonstrate promise for the diagnosis and treatment of rare diseases or conditions.
In Friday's trading, ALXN is trading at $66.14, up $2.77 or 4.37 percent on a volume of 956,290 shares.
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