Alexion submits Soliris BLA to FDA

USA-based drugmaker Alexion Pharmaceuticals says it has submitted a Biologics License Application to the Food and Drug Administration, which requests approval for use of its lead product candidate, Soliris (eculizumab), in the treatment of patients with paroxysmal nocturnal hemoglobinuria. PNH is a rare, life-threatening form of hemolytic anemia, in which the body's complement system targets and destroys red blood cells.

The BLA submission is based on data from the Phase III TRIUMPH trial, details of which were published in the most recent issue of the New England Journal of Medicine. Patients treated with Soliris in the study did not require blood transfusions, unlike those in the placebo group who needed an average of 10 during the program. In addition, 49% of the drug-treated group achieved stable hemoglobin levels, whereas none of the subjects in the placebo cohort demonstrated such stabilization.

Alexion added that it has requested a priority review for the BLA which, if granted, would expedite the agency's consideration of the drug.