Alexion’s Soliris OK’D for Blood Disorder

U.S. firm Alexion said Friday it has been OK’d to market Soliris for the rare blood disorder paroxysmal nocturnal hemoglobinuria.

PNH is characterized by chronic red blood cell destruction, or hemolysis. Soliris works by inhibiting a part of the body’s immune system called the terminal complement, which destroys the red blood cells.

PNH patients are missing an essential protein that normally keep this from happening, Alexion said. Soliris is the first so-called complement inhibitor drug approved in the United States for any disease, Alexion said.

Soliris brings real hope to people who live daily with the devastating effects of PNH, said Leonard Bell, Alexion’s CEO. With the approval of Soliris, we now have a therapy that dramatically improves the lives of patients suffering from this disease. Importantly, all patients with this life-threatening disease will be eligible for treatment.

PNH — which typically strikes patients in their early 30s — is marked by a range of symptoms including severe anemia, fatigue, recurrent pain, shortness of breath, pulmonary hypertension, and kidney disease.