FDA has approved Soliris for paroxysmal nocturnal hemoglobinuria

The FDA ( Food and Drug Administration ) has approved Soliris ( Eculizumab ).
Soliris is the first therapy approved for paroxysmal nocturnal hemoglobinuria ( PNH ), a rare, disabling and life-threatening blood disorder defined by chronic red blood cell destruction, or hemolysis. Soliris is indicated for the treatment of patients with PNH to reduce hemolysis.

Hemolysis can cause one or more of the following symptoms in patients with paroxysmal nocturnal hemoglobinuria: severe anemia, disabling fatigue, recurrent pain, shortness of breath, pulmonary hypertension, intermittent episodes of hemoglobinuria, kidney disease, impaired quality of life and thromboses.
Paroxysmal nocturnal hemoglobinuria often strikes people in the prime of their lives, with an average age of onset in the early 30's.
The estimated median survival for PNH patients is between 10 and 15 years from the time of diagnosis.

Patients with paroxysmal nocturnal hemoglobinuria are missing a specific protein that normally protects red blood cells from destruction by a component of the immune system called terminal complement. Soliris, a complement inhibitor, prevents hemolysis by selectively blocking terminal complement.

Soliris has proven to be a safe and effective therapy for paroxysmal nocturnal hemoglobinuria in three multi-national clinical studies: TRIUMPH, a placebo-controlled 26 week Phase 3 study involving 87 PNH patients, SHEPHERD, an open-label 52 week Phase 3 trial involving 97 PNH patients, and E05-001, a long term extension study.

These studies showed that Soliris reduced hemolysis in every treated patient. Hemolysis was dramatically reduced from a baseline LDH of 2,032 U/L to 239 U/L at week 26 ( p<0.001 ). The reductions in hemolysis occurred within one week of initiating treatment and were sustained for periods of up to 54 months with continued dosing of Soliris. The reduction in hemolysis expands the number of circulating PNH cells and, thereby, increases the hemoglobin level. Hemoglobin stabilization and the number of transfused packed red blood cell units, the pivotal study's co-primary endpoints, were both achieved; half of the Soliris-treated patients achieved hemoglobin stabilization compared with none of the patients in the placebo group, the median number of transfusions was reduced from 10 units/patient to 0 units/patient, respectively ( p < 0.001 in both cases ). Soliris patients reported less fatigue and improved health-related quality of life. There were fewer thrombotic events with Soliris treatment than during the same period of time prior to treatment.

Soliris is generally well tolerated. The most frequent adverse events observed in clinical studies were headache, nasopharyngitis, back pain and nausea.
Treatment with Soliris should not alter anticoagulant management because the effect of withdrawal of anticoagulant therapy during Soliris treatment has not been established.

The product label for Soliris also includes a boxed warning: " Soliris increases the risk of meningococcal infections. Vaccinate patients with a meningococcal vaccine at least 2 weeks prior to receiving the first dose of Soliris; revaccinate according to current medical guidelines for vaccine use. Monitor patients for early signs of meningococcal infections, evaluate immediately if infection is suspected, and treat with antibiotics if necessary. "
Two out of 196 vaccinated PNH patients treated with Soliris experienced a serious meningococcal infection.

Source: Alexion, 2007


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