Long-Acting Complement Inhibitor Gets Priority Review for PNH
A Prescription Drug User Fee Act (PDUFA) target date of February 18, 2019 has been set by the FDA for the PNH indication.
Immune-Mediated Blood Disorders
AAP Revise Policy Statement on Cord Blood Banking
Physicians need to be able to explain concepts of autologous and allogeneic use of cord blood units, as well as benefits and limitations of blood banking and transplantation.
Investigational PNH Treatment Granted Orphan Drug Status
“We have designed RA101495 for once-daily, subcutaneous self-administration, an approach which has the potential to ease this burden, improve convenience, and provide much-needed dosing flexibility,” said Doug Treco, PhD, President and CEO of Ra Pharma.
Investigational PNH Treatment Granted Orphan Drug Status
“We have designed RA101495 for once-daily, subcutaneous self-administration, an approach which has the potential to ease this burden, improve convenience, and provide much-needed dosing flexibility,” said Doug Treco, PhD, President and CEO of Ra Pharma.
Coversin Gets Fast Track Status for Paroxysmal Nocturnal Hemoglobinuria
Akari is currently investigating Coversin in two Phase 2 clinical trials in patients with paroxysmal nocturnal hemoglobinuria.
ACOG Updates Recommendations on Carrier Screening
Ethnic-specific, panethnic, and expanded carrier screening are all acceptable screening strategies.
Gammaplex 10% Gets FDA Approval
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
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
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
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.
