Pharmaceutical Industry discussion, review, analysis, news and information.

The USFDA has recently approved Soliris™ (eculizumab) for the treatment of all patients with paroxysmal nocturnal hemoglobinuria (PNH).

A product of Alexion Pharmaceuticals, Inc., (Nasdaq: ALXN), Soliris™ is the first therapy ever approved for PNH and is indicated for the treatment of patients with PNH to reduce hemolysis.

PNH or paroxysmal nocturnal hemoglobinuria is a rare, disabling and life-threatening blood disorder defined by chronic red blood cell destruction, or hemolysis – causing one or more of the following symptoms in patients with PNH: severe anemia, disabling fatigue, recurrent pain, shortness of breath, pulmonary hypertension, intermittent episodes of dark colored urine (hemoglobinuria), kidney disease, impaired quality of life and blood clots (thromboses). (1,2)

Patients with PNH are missing a specific protein that normally protects red blood cells from destruction by a component of the immune system called terminal complement. Soliris, the first complement inhibitor approved in the United States for the treatment of any disease, prevents hemolysis by selectively blocking terminal complement.

According to Leonard Bell, MD, chief executive officer of Alexion Pharmaceuticals:

"Soliris brings real hope to people who live daily with the devastating effects of PNH. 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.”

Find more details from the full report.

(1) Rother R, Bell L, Hillmen P, Gladwin M. The clinical sequelae of intravascular hemolysis and extracellular plasma hemoglobin. JAMA 2005; 293:1653-1662.

(2) Parker C, Omine M, Richards S, et al. Diagnosis and management of paroxysmal nocturnal hemoglobinuria. Blood 2005; 106:3699-3709.