The U.S. Food and Drug Administration (FDA) recently granted orphan drug status to SHP654 (BAX 888), an investigational factor VIII (FVIII) gene therapy candidate being developed by Shire for the treatment of hemophilia A. Gene therapy allows the delivery of a functional copy of the defective gene.
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Jivi was safe and effective at preventing and treating bleeds for more than five years in boys under age 12 with severe hemophilia A, according to long-term data from a Phase 3 trial’s extension study. Data analysis also showed that most of…
Geneventiv Therapeutics said it had a “positive and constructive” meeting with the U.S. Food and Drug Administration (FDA) on its plan to develop GENV-HEM, a gene therapy for hemophilia A. The meeting “represents a key regulatory milestone for Geneventiv,” Damon Race, CEO of Geneventiv, said in a…
Precision Biologic has announced an update of its Cryocheck Chromogenic Factor VIII assay, a test used to help diagnose and manage hemophilia A. The update, which is expected to be commercially available in the coming months, will ensure that the assay’s accuracy is not compromised by a new…
When I have the opportunity to gather with my brothers and sisters from the hemophilia community, I notice we all have stories to tell. When you live with a rare disease, you are bound to have unique interactions with medical providers, family members, friends, and colleagues. These interactions range from…
Extended half-life (EHL) blood-clotting factor products are safe and effective at preventing bleeds during minor and major surgeries in people with hemophilia, according to a real-world study in Nordic countries. While dosing recommendations in the perioperative period, or the period around surgery, vary across Nordic hemophilia treatment centers…
Testing levels of thrombin — an enzyme that helps blood clot — in people with acquired hemophilia A could help doctors fine-tune care for these patients when both clot-promoting and blood-thinning medications are used. That’s the conclusion drawn by researchers in the Netherlands, who found that thrombin testing in…
A liver transplant can be live-saving for hemophilia patients, but may not always fully cure the disease, according to the case report about a hemophilia A patient. The letter to the editor describing the report, “Orthotopic liver transplantation for haemophilia A may not…
A single dose of Spark Therapeutics’ investigational gene therapy SPK-8011 increased the levels of factor VIII (FVIII) — the missing clotting protein in hemophilia A — and, in 16 of 18 male patients, those levels were sustained for up to four years, according to results from a Phase 1/2 clinical…
Spontaneous and joint bleeds, and limits on physical activity are evident in people with severe hemophilia AÂ even when they adhere to and remain on preventive treatment, an analysis of global, real-world data showed. These findings highlight the need for additional alternative therapies for patients with severe disease. The study,…