Bleeding Disorders
News
FDA Approves Pfizer’s Hympavzi for Hemophilia A, B
New weekly injection treatment option to prevent or reduce bleeding in hemophilia A and B for patients aged 12 and older gets FDA approval.
Feature
Pfizer’s Withdrawal of SCD Drug Raises Questions
The worldwide withdrawal by Pfizer of its sickle cell disease drug voxelotor has hematologists wondering why and racing to inform their patients...
Conference Coverage
No Matched Sibling Donor? Sickle Cell Experts Debate Next-Best Option
SCD specialists differ on the best treatment choices for patients who lack an available matched sibling donor.
News
Two Soliris Biosimilars Approved for PNH in the US
For treatment of two rare blood disorders, the FDA has just greenlighted two monoclonal antibodies.
From the Journals
Hemophilia: Novel Tx Also Cuts Bleeding in Kids
For pediatric patients with hemophilia A, once-weekly factor VIII prophylaxis shows bleeding protection similar to that for adults.
Sponsored Supplement
Best Practices: In the Management of Hemophilia
Conference Coverage
Hemophilia: Marstacimab Sustains Long-Term Bleeding Reduction
In an interim analysis, this novel, weekly subcutaneous injection shows continued benefits in the prevention of bleeding in hemophilia A and B.
Feature
Beta Thalassemia: Pricey Gene Therapy Hits The Mark
A landmark new treatment for bleeding disorder banishes the need for transfusions, but it’s exorbitantly expensive.
From the Journals
SCD: Delaying Transition to Adult Care Poses Risks
More emergency visits and greater risks of hospitalizations if young patients with sickle cell delay their transition to adult care.
News
FDA Approves Second Gene Therapy for Hemophilia B
For adults with hemophilia B, a second gene therapy — also priced at $3.5 million — has been greenlighted by the Food and Drug Administration.
News
FDA OKs Danicopan Add-On for Extravascular Hemolysis in Adults With PNH
Danicopan to support treatment of a rare blood disorder, paroxysmal nocturnal hemoglobinuria, is approved by the Food and Drug Administration.
News
EU Backs First Oral Monotherapy for Adults With PNH
The European Medicines Agency has greenlighted the sale of Fabhalta for adult patients with a rare bleeding disorder.