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Final Patient Treated in Phase 3 Trial of Eculizumab in Patients With Paroxysmal Nocturnal Hemoglobinuria
Approximately 85 PNH patients were randomized into the six month treatment phase in TRIUMPH, which exceeded the patient requirements agreed upon with the U.S. Food and Drug Administration (FDA) as part of the Special Protocol Assessment (SPA) process. Having completed treatment of the final patient, it is anticipated that top line clinical results will be available later in the first quarter of this year.
"We are pleased to have completed the final protocol-specified treatment in the TRIUMPH trial, on target with our earlier announced schedule," said Leonard Bell, M.D., Chief Executive Officer of Alexion. "Additionally, we are encouraged that every eligible TRIUMPH patient to date has opted to enroll in an open-label extension trial to receive eculizumab. We are keenly focused on progressing our internal efforts to compile our regulatory applications for submission, pending clinical results, to U.S. and European regulatory authorities later this year to market eculizumab for use in PNH patients."
TRIUMPH is a double-blind, randomized, placebo-controlled multi-center pivotal Phase III trial, examining the effects of eculizumab on the co-primary endpoints of hemoglobin stabilization and blood transfusion requirement in hemolytic, transfusion-dependent PNH patients during six months of therapy. The study enrolled patients in the US, Canada, Europe, and Australia. TRIUMPH is designed to be a single pivotal efficacy trial for eculizumab therapy in PNH.
In addition to TRIUMPH, the pivotal Phase III clinical program includes the SHEPHERD trial. SHEPHERD is an open-label, non-placebo-controlled, multi-center clinical trial primarily aimed at generating additional safety data with eculizumab in a broader population of hemolytic PNH patients with a history of transfusions. Efficacy measures will also be obtained during the study. The SHEPHERD protocol includes 12 months of treatment with a six month interim analysis.
Alexion previously reached an agreement with the FDA on the design of TRIUMPH and SHEPHERD under the FDA's SPA process. It is expected that, if successful, the combined trials will comprise the application that will serve as the primary basis of review for the approval of a Biologics License Application (BLA) for eculizumab in the PNH indication. Pending analyses of results from TRIUMPH and SHEPHERD and subsequent discussions with U.S. and European regulatory authorities, Alexion anticipates submitting applications later this year to both U.S. and European regulatory authorities for approval to market eculizumab for use in PNH patients.
PNH is a rare blood disorder characterized by the onset of severe hemolytic anemia, chronic fatigue and intermittent episodes of dark colored urine, known as hemoglobinuria. PNH patients are also at increased risk of forming life-threatening blood clots, or thromboses, which are a leading cause of death in this disease. People with PNH have an acquired deficiency of proteins that normally protect red blood cells from a component of the body's natural defense system, known as the complement cascade. Lack of these complement inhibitor proteins leaves PNH red blood cells susceptible to destruction (hemolysis) by terminal complement, causing patients to become anemic. Eculizumab is designed to block production of terminal complement, thereby preventing PNH red blood cell destruction. There currently is no drug specifically available for treatment of patients with PNH.
Based upon scientific investigations and presentations of the prevalence of patients diagnosed with abnormal PNH cells in their blood, it is currently estimated that approximately 8,000 - 10,000 people in North America and Western Europe may suffer from PNH.
Source: Alexion Pharmaceuticals, Inc.