Bleeding Disorders
News
FDA grants priority review to NDA for avatrombopag
The US Food and Drug Administration (FDA) has granted priority review to the new drug application (NDA) for avatrombopag. Avatrombopag is a...
News from the FDA/CDC
FDA grants orphan drug status to rofecoxib for hemophilic arthropathy
An nonopioid treatment for hemophilic arthropathy could be on the way.
News
FDA approves first-in-class drug for hemophilia A with Factor VIII
The new treatment is given as a weekly injection.
News
Product approved to treat patients with hemophilia A and inhibitors
The US Food and Drug Administration (FDA) has approved use of emicizumab-kxwh (Hemlibra®), a bispecific factor IXa- and factor X-directed antibody...
News
CHMP backs therapy for hemophilia A
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended marketing authorization for rurioctocog alfa...
News
CHMP recommends approval of romiplostim in kids
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended expanding the approved indication for...
News
Drug receives orphan designation for treatment of PNH
The US Food and Drug Administration (FDA) has granted orphan drug designation to ACH-4471 for the treatment of paroxysmal nocturnal hemoglobinuria...
Feature
Sickle cell patients suffer discrimination, poor care – and shorter lives
Sickle cell disease is “a microcosm of how issues of race, ethnicity and identity come into conflict with issues of health care.”
News
Product can improve joint health in hemophilia A
New research suggests prophylaxis with a recombinant factor VIII Fc fusion protein (rFVIIIFc) can improve joint health over time in patients with...
News
Hemophilia B therapy approved in Saudi Arabia
The Saudi Food and Drug Authority has approved eftrenonacog alfa (Alprolix®) for the treatment of hemophilia B in the Kingdom of Saudi Arabia....
News
Gene therapy granted orphan designation for hemophilia A
The US Food and Drug Administration (FDA) has accepted an investigational new drug application for SHP654 (also known as BAX 888) and granted the...