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.
A Prescription Drug User Fee Act (PDUFA) target date of February 18, 2019 has been set by the FDA for the PNH indication.
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.
“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.
“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.
Akari is currently investigating Coversin in two Phase 2 clinical trials in patients with paroxysmal nocturnal hemoglobinuria.
Ethnic-specific, panethnic, and expanded carrier screening are all acceptable screening strategies.
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.