Novo Nordisk Halts Concizumab Trials After Thrombotic Events
After observing blood clots in some patients enrolled in clinical trials of concizumab, Novo Nordisk has paused one mid-stage and two late-stage studies of...
Sickle Cell Disease Gene Therapy Receives Orphan Drug Designation
ARU-1801, an investigational gene therapy for the treatment of sickle cell disease (SCD) and beta thalassemia, was granted orphan drug designation by the FDA.
ARU-1801...
BioMarin Sets High Price Tag for Hemophilia Gene Therapy Candidate
If its experimental gene therapy for patients with hemophilia A is approved by the FDA, BioMarin Pharmaceutical is considering pricing valoctocogene roxaparvovec (valrox) between...
ICER Report Finds That Sickle Cell Drugs Are Too Expensive
According to a draft report by the Institute for Clinical and Economic Review (ICER), SCD drugs made by Novartis, Global Blood Therapeutics (GBT), and...
How Common Is Serotonin Release Assay-Negative Heparin-Induced Thrombocytopenia?
Heparin-induced thrombocytopenia (HIT), a prothrombotic drug reaction caused by platelet-activating anti-platelet factor 4 (PF4)/heparin antibodies, is commonly diagnosed with the platelet serotonin-release assay (SRA)...
FDA Approves Ferric Derisomaltose for Iron-Deficiency Anemia
The ferric derisomaltose injection received FDA approval for the treatment of iron-deficiency anemia in adults who are intolerant to oral iron, whose disease has...
Evaluating Efgartigimod in Patients With Primary Immune Thrombocytopenia
Treatment with the neonatal Fc receptor antagonist efgartigimod led to clinically relevant increases in platelet counts in patients with primary immune thrombocytopenia (ITP), according...
FLT180a Shows Efficacy in Hemophilia B at High Doses
When administered at a high dose, the experimental gene therapy FLT180a restored activity of clotting factor IX (FIX) within normal ranges in men with...
ER-Based Protocol Improves the Accuracy of von Willebrand Disease Testing in Young Patients With...
Watch our interview with Megan C. Brown, MD.
Researchers at Children’s Healthcare of Atlanta (CHOA) have developed an emergency department (ED)–based protocol that helps standardize...
The High Price of Hemophilia
An estimated 20,000 people in the U.S. are living with the inherited bleeding disorder hemophilia, and more than 60% of them have moderate or...
Current Issue
March 2020, Volume 6, Issue 4
This issue looks at how cost-effective analyses are used to inform health-care decisions, explains the latest maintenance of certification requirements, and more.
