More than 21 years ago, when my first child was born and diagnosed with severe hemophilia A, he was very fortunate to have first-generation clotting factors available. These were concentrates stabilized with albumin that were very safe. When the factor was reconstituted with sterile water, a seemingly…
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The U.S. Food and Drug Administration (FDA) has pushed back by more than one year a decision on Roctavian, the potential first gene therapy for adults with hemophilia A. In a complete response letter that took the therapy’s developer, BioMarin by surprise, the FDA said it needed…
Infection and related complications pose a serious risk to people with acquired hemophilia A (AHA), accounting for more than half of the deaths in a registry study in Spain. About 15% of participants in the study experienced at least one infection requiring treatment or hospitalization, the study found. About…
Health Canada recently authorized Shire Pharma Canada‘s treatment Adynovate for hemophilia A patients younger than 12 based on results of a Phase 3 clinical trial. Approved Nov. 21, the injectible factor replacement therapy is available to pediatric patients for the prevention and control of bleeding episodes, as well…
Hemophilia is a rare disorder in which a person’s blood is not able to clot properly, resulting in unusually easy and prolonged bleeding.
Treatment of hemophilia A with bypassing agents is associated with significantly higher costs than with factor replacement therapy, according to a real-world study in the U.S. The study, “Healthcare costs among patients with hemophilia A treated with factor replacement or bypassing agents,” appeared in…
Hemophilia is a genetic disorder that affects your blood’s ability to clot. Hemophilia sufferers lack a clotting factor, meaning they have an increased risk of bruising and bleeding, both externally and internally around joints and in the brain. MORE: Learn more about hemophilia treatment options. There are three levels of…
A single dose of the gene therapy Roctavian (valoctocogene roxaparvovec-rvox) continues to prevent bleeds and the need for preventive treatment (prophylaxis) for up to five years in most treated men with severe hemophilia A, according to final Phase 3 trial results. Activity of factor VIII (FVIII), the clotting…
Humate-P (human antihemophilic factor/von Willebrand factor complex) is a plasma-derived factor replacement therapy approved to prevent and treat bleeding episodes in people with hemophilia A.
An experimental therapy may help remove harmful antibodies that reduce the effectiveness of factor replacement therapies in hemophilia A, potentially offering a new way to overcome one of the most significant complications of standard care. The therapy uses chimeric autoantibody receptor T cells, or CAAR-T cells. These are…