Adults across the general public in Canada think an under-the-skin injection treatment for hemophilia A would be preferable to those needing to be infused intravenously, or directly into a vein, a study based on interviews reported. Results of the analysis, sponsored by Roche Canada and conducted by Broadstreet…
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Continuous into-the-vein (intravenous) infusions of Advate immediately before and after major orthopedic surgery is as safe and effective as standard, short, bolus infusions at preventing bleeds in men with moderate-to-severe hemophilia A. That finding from a Phase 3/4 clinical trial — the first controlled trial to compare the two types…
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Advantages, Unanswered Questions About PEGylated Products for Hemophilia A Reviewed by Researchers
Researchers reviewed the current hemophilia research that focuses on the development of improved longer-acting factor replacement therapies, particularly PEGylated products. The new clinical research advances lead the scientific community to look at such developments with optimism, but also some caution. The study, “Potential role of a new PEGylated recombinant…
I don’t think anyone likes having a chronic disease. I have good days, and I have bad days. Thankfully, the good days outnumber the bad ones. There are days when I tolerate having hemophilia, days when I can step back and see the gifts that have come from…
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…
Prescriptions for extended half-life products to hemophilia A patients in Japan increased from 2016 to 2020, according to a new study that analyzed real-world data from a hospital database. This was accompanied by an increase in healthcare costs seen for patients switching from standard half-life factor replacement therapies to…
The European Medicines Agency (EMA) Pharmacovigilance Risk Assessment Committee (PRAC) has initiated a review of factor VIII-containing medicines, to evaluate the risk of inhibitor protein development in patients starting treatment for hemophilia A, according to a press release. This review, which will cover all medicines containing factor VIII authorized in the…
Treatment with Esperoct led to stabilization and/or improvement of health-related quality of life (HRQoL) in patients with severe hemophilia A from all age groups, a study suggests. The study, “Health-related quality-of-life and treatment satisfaction of individuals with hemophilia A treated with turoctocog alfa pegol (N8-GP): a new recombinant…
Biomarin Pharmaceutical is looking to sell off rights to Roctavian (valoctocogene roxaparvovec-rvox), its approved gene therapy for hemophilia A. “As we focus on the business units aligned with our strategic priorities, today we are announcing the decision to pursue options to divest Roctavian and remove it from…
Investigational gene therapy SB-525 is well-tolerated and leads to a dose-dependent increase in factor VIII (FVIII), according to interim results from eight patients with severe hemophilia A enrolled in the Phase 1/2 Alta clinical trial. Data showed that patients who received the highest dose of SB-525 — 3e13 vg/kg dose — reached…