For the first time, as part of a U.S. clinical trial, the cell therapy BE-101 has been administered to someone with hemophilia B. Be Biopharma, the company developing BE-101, announced the dosing of the first participant in a first-in-human Phase 1/2 clinical trial called BeCoMe-9 (NCT06611436). The…
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For the first time in Denmark, people with hemophilia B have been treated with the gene therapy Hemgenix (etranacogene dezaparvovec), according to an announcement from CSL Behring, the company that markets the therapy. Hemgenix was cleared for use in the European Union in early 2023, following its…
Two potential gene therapies — AMT-060 and AMT-061 (etranacogene dezaparvovec) — lead to safe and long-term increases in factor IX (FIX) activity, as well as fewer bleeds among patients with hemophilia B, according to the therapies’ maker, uniQure. The company announced in a press release that…
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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…
FLT180a, also known as verbrinacogene setparvovec, is an investigational gene therapy that was investigated as a treatment for people with hemophilia B.
Switching to a different clotting factor VIII (FVIII) product was not linked to a greater risk of developing neutralizing antibodies against FVIII, or FVIII inhibitors, in people with hemophilia A, a recent study reported. Changing treatment also had no impact on a patient’s immune profile or on therapy efficacy.
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CSL Behring’s Afstyla Allows Hemophilia A Patients to Use Less Factor Replacement, Analysis Shows
Hemophilia A patients treated with CSL Behring’s Afstyla (recombinant factor 8 single-chain) require less factor to control bleeding compared to those taking octocog alfa, the company recently announced. The lower consumption holds both for patients using clotting factors on-demand and those who require preventive, or prophylactic treatment. CSL Behring…
The Spanish Interministerial Commission on the Pricing of Medicines, known as CIPM, has issued a positive recommendation for national reimbursement of Hemgenix (etranacogene dezaparvovec) for eligible people with hemophilia B, according to CSL Behring, the company that markets the gene therapy. The recommendation means that patients in Spain should…
Hemophilia is a rare disorder in which a person’s blood is not able to clot properly, resulting in unusually easy and prolonged bleeding.
Directly switching from Hemlibra (emicizumab) to Novo Nordisk‘s investigational Mim8 (denecimig) was well tolerated in adults and adolescents with hemophilia A, regardless of whether they had inhibitors or not. That’s according to results from the now-completed Phase 3b FRONTIER5 study (NCT05878938), which assessed the safety of switching…