1993) Deficiency of the GPI anchor caused by a somatic mutation of the PIG-A gene in paroxysmal nocturnal hemoglobinuria
Paroxysmal nocturnal hemoglobinuria
(PNH) is a rare, acquired clonal hematopoietic stem cell disorder, which is caused by a somatic mutation in the phosphatidylinositol glycan-complementation class A gene, with an estimated prevalence of 1-2 cases per million people in the United States (1-2).
Clinical history and laboratory studies compatible with intravascular hemolysis, including that caused by mechanical hemolysis from a dysfunctional cardiac valve replacement as in this case or paroxysmal nocturnal hemoglobinuria
, aid with diagnosis.
Soliris, a designated orphan medicinal product, is intended to reduce hemolysis (destruction of red blood cells) in patients with paroxysmal nocturnal hemoglobinuria
(PNH), The CHMP recommended a broad label for Soliris covering all patients with PNH.
STUDY DATA: Alexion announced that the pivotal Phase 3 study of ALXN1210, the company's investigational long-acting C5 complement inhibitor, demonstrated non-inferiority to Soliris in complement inhibitor treatment-naive patients with paroxysmal nocturnal hemoglobinuria
based on the co-primary endpoints of transfusion avoidance and normalization of lactate dehydrogenase levels, a direct marker of complement-mediated hemolysis in PNH.
The company has developed and commercializes the first and only approved complement inhibitor to treat patients with paroxysmal nocturnal hemoglobinuria
and atypical hemolytic uremic syndrome.
Soliris is the first and only treatment for patients with Paroxysmal Nocturnal Hemoglobinuria
, a rare and life-threatening blood disorder characterized by excessive destruction of red blood cells; and Atypical Hemolytic Uremic Syndrome, a rare and life-threatening genetic disorder characterized by blood clots in small vessels.
Biopharmaceutical company Akari Therapeutics (NasdaqGS:AKTX) reported on Thursday the receipt of US Food and Drug Administration (FDA) fast frack designation for Coversin for treatment of paroxysmal nocturnal hemoglobinuria
(PNH) in patients who have polymorphisms conferring eculizumab resistance.
A review of the patient's medical records revealed a history of paroxysmal nocturnal hemoglobinuria
and current biweekly eculizumab (Soliris) therapy.
GlobalData's clinical trial report, "Paroxysmal Nocturnal Hemoglobinuria
Global Clinical Trials Review, H2, 2015" provides an overview of Paroxysmal Nocturnal Hemoglobinuria
clinical trials scenario.
Alexion developed Soliris (eculizumab) from the laboratory through regulatory approvals, and currently provides Soliris to patients around the world with paroxysmal nocturnal hemoglobinuria
and atypical hemolytic uremic syndrome, two life-threatening ultra-rare disorders.
com)-- Access Health[R], a new magazine-style health and wellness series will dedicate an entire 30-minute episode to the rare blood disease PNH, which stands for Paroxysmal Nocturnal Hemoglobinuria
, a rare and potentially life-threatening disease, on Wednesday, February 11 at 7:30 a.