SPK-8016, also known as RG6358, is an experimental gene therapy that was being investigated for hemophilia A patients who had developed neutralizing antibodies (inhibitors) against clotting factor VIII (FVIII).
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Hemophilia A treatments include preventive therapies like Hemlibra, which can lower patient expenses, and Wilate, a plasma-derived replacement therapy. Additionally, low-dose recombinant factor VIII effectively prevents bleeding in children. For gene therapies, BMN 270 clinical trials resumed enrollment, while SPK-8016 development was discontinued.
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The Medicines and Healthcare Products Regulatory Agency (MHRA) for the U.K. has approved the resumption of patient enrollment in BioMarin Pharmaceuticals’ Phase 1/2 clinical trial evaluating the investigational gene therapy BMN 270 as a treatment for severe hemophilia A. BioMarin suspended patient dosing in the trial in June because of increasing levels…
Starting or switching to prophylactic, or preventive Hemlibra (emicizumab) may lead to a significant reduction in treatment-associated expenses for hemophilia A patients in the U.S., a real-world study reported. These findings highlight the cost-benefits of using Hemlibra — and may help future cost analysis studies in assessing new…
Wilate (human von Willebrand factor/coagulation factor VIII complex) is a plasma-derived replacement therapy that's approved to prevent and control bleeds in adults and adolescents with hemophilia A. It is given via an into-the-vein, or intravenous, injection.
Low-dose recombinant factor VIII lowered bleeding rates in Chinese children with hemophilia, and the earlier the preventive treatment began, the more effective it was in minimizing bleeding episodes, according to a new study. The research, “The Efficacy of Recombinant FVIII Low-Dose Prophylaxis in Chinese Pediatric Patients With Severe Hemophilia A:…
Nuwiq (simoctocog alfa) is an approved therapy that works to prevent and control bleeding episodes, including those occurring during surgery, in adults and children with hemophilia A.
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…
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…
The European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended marketing authorization for Afstyla (lonoctocog alfa) for the treatment and prevention of bleeding in adults and children with hemophilia A. Developed by CSL Behring, Afstyla is a single-chain recombinant human factor VIII…