Immune-mediated Blood Disorders

Coversin Gets Fast Track Status for Paroxysmal Nocturnal Hemoglobinuria

By March 31, 2017

Akari is currently investigating Coversin in two Phase 2 clinical trials in patients with paroxysmal nocturnal hemoglobinuria.

ACOG Updates Recommendations on Carrier Screening

February 27, 2017

Ethnic-specific, panethnic, and expanded carrier screening are all acceptable screening strategies.

Gammaplex 10% Gets FDA Approval

By February 07, 2017

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.

Life-Threatening Blood Disorder Treatment Gets Orphan Drug Status

By January 06, 2017

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).

FDA Fast Tracks Rare Hematological Disorder Treatment

By December 20, 2016

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)

FDA Grants Orphan Drug Status to Coversin for Rare Blood Disorder

By September 13, 2016

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.

Luspatercept Fast Tracked for Two Beta-Thalassemia Indications

By May 18, 2015

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.

First Pathogen Reduction System to Prep Plasma, Platelets for Transfusion Approved

By December 17, 2014

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).

Octagam 10% Now Available for Chronic Immune Thrombocytopenic Purpura

October 27, 2014

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).

Investigational Sickle Cell Disease Agent Fast Tracked

October 02, 2014

The Food and Drug Administration (FDA) has granted Fast Track designation to NKTT120 for the treatment of sickle cell disease.

mTORC Pathway Involved in Antiphospholipid Sx Vasculopathy

July 24, 2014

The mammalian target of rapamycin complex (mTORC) pathway seems to be involved in antiphospholipid syndrome-associated vascular lesions.

Busulipo Designated Orphan Drug for Bone Marrow Transplant

October 29, 2013

The FDA has designated Busulipo (MZH-2; Pharmalink) Orphan Drug status for use in cancer patients prior to hematopoietic stem cell transplantation (HSCT).

Biomarker IDs Tx-Resistant Graft-Versus-Host Disease

August 08, 2013

Researchers found that levels of suppression of tumorigenicity 2 (ST2) were most significantly associated with outcome.

Rituximab Benefits Some APS Non-Criteria Manifestations

December 10, 2012

Some patients with antiphospholipid syndrome with non-criteria manifestations may improve after treatment with rituximab.

Infliximab + Standard IVIG Reduces Inflammation in Kawasaki Disease

By October 21, 2012

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.

Shelf life of Hizentra extended from 18 to 24 months

August 18, 2010

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.

GammaGard Liquid recalled

June 07, 2010

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]).