Eculizumab (Soliris) Therapy For Rare Blood Disease

Blood Disease

Alexion Pharmaceuticals, Inc., has received marketing approval from the FDA for eculizumab (Soliris). This is the first therapy approved for paroxysmal nocturnal hemoglobinuria  (PNH), a rare, life-threatening blood disorder characterized by chronic red blood cell destruction (hemolysis).

In patients with this acquired genetic blood disorder, the red blood cells are destroyed by complement, a component of the body’s immune system. PNH affects 8,000 to 10,000 people in North America and Europe.

Hemolysis can cause severe anemia, blood clots, disabling fatigue, recurrent pain, shortness of breath, pulmonary hypertension, dark-colored urine, kidney disease, and impaired quality of life.

The product label includes a boxed warning of an increased risk of meningo-coccal infections. Patients should be vaccinated with a meningococcal vaccine at least two weeks before they receive the first dose of eculizumab. canada drugs pharmacy

Before beginning therapy, all patients and their physicians will be enrolled in the Soliris Safety Registry, part of a risk-management program.

Pre-approval trials of eculizumab are discussed in this month’s Meeting Highlights feature.