PK-guided prophylaxis reduces dosing, still prevents bleeds

Antihemophilic factor
Personalized prophylaxis with a recombinant factor VIII product allowed for reduced dosing while still providing protection from bleeds in a small study of patients with severe hemophilia A. Researchers tested pharmacokinetic (PK)-guided prophylaxis with simoctocog alfa in previously treated patients with severe hemophilia A. The approach reduced the dose administered and... [Read Article]

Bivalirudin not superior to heparin in real-world analysis

Vial of heparin
A new study suggests bivalirudin does not produce better outcomes than heparin in patients undergoing transradial primary percutaneous coronary intervention (PCI) for ST elevation myocardial infarction (STEMI). Researchers found no significant difference in the rate of a composite of death, myocardial infarction, and stroke in patients who received bivalirudin or... [Read Article]

EMA recommends drug receive orphan designation for PNH

Red blood cells
The European Medicines Agency (EMA) has recommended orphan drug designation for the complement C3 inhibitor APL-2 as a treatment for paroxysmal nocturnal hemoglobinuria (PNH). APL-2 is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b, blocking all 3 pathways of complement activation... [Read Article]