Immune-mediated Blood Disorders
Akari is currently investigating Coversin in two Phase 2 clinical trials in patients with paroxysmal nocturnal hemoglobinuria.
Bio Products Laboratory announced that the Food and Drug Administration (FDA) has approved Gammaplex 10% (immune globulin intravenous [human], 10% liquid) for the treatment of primary immunodeficiency (PI) and chronic immune thrombocytopenic purpura (ITP) in adults.
Alexion has announced that the Food and Drug Administration (FDA) has granted orphan drug designation to ALXN1210, their candidate treatment for paroxysmal nocturnal hemoglobinuria (PNH).
Apellis has announced that the Food and Drug Administration (FDA) has granted Fast Track designation to APL-2, a novel drug candidate studied for the treatment of paroxysmal nocturnal hemoglobinuria (PNH)
The FDA has granted Orphan Drug designation to Coversin (Akari Therapeutics) for the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH), an ultra-rare, life-threatening and debilitating hematological disorder.
The Food and Drug Administration (FDA) has granted Fast Track designations to luspatercept (Celgene and Acceleron) for use in two separate indications: for the treatment of patients with transfusion dependent beta-thalassemia and for the treatment of patients with non-transfusion dependent beta-thalassemia.
The Food and Drug Administration (FDA) has approved Cerus' Intercept Blood System for plasma and platelets, the first pathogen reduction system to use in preparation of plasma and platelets in order to reduce the risk of transfusion-transmitted infections (TTI).
Octapharma USA announced that Octagam 10% [Immune Globulin Intravenous (Human)] liquid preparation is now available for the treatment of adults with chronic immune thrombocytopenic purpura (ITP).
The Food and Drug Administration (FDA) has granted Fast Track designation to NKTT120 for the treatment of sickle cell disease.
The mammalian target of rapamycin complex (mTORC) pathway seems to be involved in antiphospholipid syndrome-associated vascular lesions.
The FDA has designated Busulipo (MZH-2; Pharmalink) Orphan Drug status for use in cancer patients prior to hematopoietic stem cell transplantation (HSCT).
Adding infliximab to standard intravenous immunoglobulin to reduce inflammation in acute Kawasaki disease is safe in children, even those younger than 1 year, according to a phase 3 randomized trial presented at IDWeek 2012.
The FDA has approved a supplemental Biologics License Application (sBLA) to extend the shelf life of Hizentra (immune globulin subcutaneous [Human] 20% Liquid, from CSL Behring) from 18 to 24 months.
The FDA and Baxter BioScience have notified healthcare professionals of a voluntary market withdrawal being conducted as a precautionary measure due to an increased number of adverse event reports of allergic reactions associated with two lots of GammaGard Liquid (immune globulin intravenous [human]).