Alexion Pharmaceuticals, Inc. (ALXN) Tuesday said the European Commission extended the therapeutic indication for Soliris to include the treatment of pediatric and adult patients with atypical hemolytic uremic syndrome or aHUS. Alexion said Soliris is the first therapy approved in the European Union and the U.S. to treat the ultra-rare, life-threatening disease.
In September, the U.S. Food and Drug Administration has approved Soliris for the treatment of aHUS in adults and children.
Atypical hemolytic uremic syndrome progressively damages vital organs, leading to stroke, heart attack, kidney failure and death. The abnormal activation of the pathogen-clearing system results in the formation of multiple blood clots in small blood vessels throughout the body, known as thrombotic microangiopathy or TMA.
Soliris, a first-in-class terminal complement inhibitor, specifically targets uncontrolled complement activation. In clinical studies, chronic treatment with Soliris resulted in a rapid and sustained reduction in complement-mediated TMA.
In study C08-003 A/B, which included 20 patients with a long duration of aHUS and prior treatment before starting on Soliris, 16 out of 20 or 80 percent Soliris-treated patients achieved TMA event-free status.
In study C08-002 A/B, which included 17 patients with progressive clinical TMA complications despite intensive PE/PI, Soliris inhibited complement-mediated TMA as shown by a significant improvement in platelet count from baseline through week 26.
Study C009-001r included 15 pediatric patients (ages 2 months to less than 12 years) who received Soliris outside of prospective clinical trials and with or without prior PE/PI. Platelet count was normalized in 14 of 15 Soliris-treated patients.
Alexion expects to start serving patients with aHUS in initial major European countries in the first half of 2012, with additional major European countries commencing through mid-2013.
Soliris is also approved in the United States, European Union, Japan and other territories for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH), a debilitating, ultra-rare and life-threatening blood disorder.
ALXN is currently trading at $65.84, down $1.63 or 2.42%, on the Nasdaq. Over the past year, the stock traded in a range of $37.47 - $70.42.
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