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…
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Clinical trials are evaluating various hemophilia treatments, including gene therapy for severe hemophilia A, dalcinonacog alfa for hemophilia B, and TRM-201 for hemophilic arthropathy. MarzAA also shows potential for preventing bleeding in hemophilia A or B patients with inhibitors.
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Catalyst Biosciences announced the beginning of enrollment for a Phase 2b clinical trial for dalcinonacog alfa (DalcA) in people with Hemophilia B. Hemophilia B is caused by the lack of a functional Factor IX clotting protein. Like many available therapeutics for Hemophila B, DalcA is essentially a lab-manufactured version…
Key findings from clinical trials of three recombinant coagulation factors as new hemophilia treatments, presented at last year’s Congress of the International Society on Thrombosis and Haemostasis (ISTH 2015), have been published in a supplemental issue of the journal Thrombosis Research. The results were presented in a session…
Funding has been set for the RESET-HA Phase 3 clinical trial evaluating Tremeau Pharmaceuticals‘ TRM-201 (rofecoxib) in people with hemophilic arthropathy — a painful and degenerative disease caused by recurrent bleeding in the joints. The private investment firm Gurnet Point Capital also will support other aspects of TRM-201‘s…
Clinical data from the ongoing Phase 2/3 trial data evaluating MarzAA (marzeptacog alfa activated) in patients with Hemophilia A or B with inhibitors continues to demonstrate the treatment’s potential to prevent bleedings. The most recent trial results were discussed at the 12th Annual Congress of the European Association for Haemophilia…
Zepatier (elbasvir/grazoprevir) is an effective and safe treatment for hepatitis C infection in hemophilia patients and others with inherited blood disorders — a group that has been particularly exposed to such infection, according to a Phase 3 trial that examined the drug specifically in this patient group. The study,…
Two clinical trials launching in December will use Octapharma USA’s Wilate, von Willebrand Factor/Coagulation Factor VIII Complex (Human) Lyophilized Powder for Solution for Intravenous Injection, in patients with hemophilia A. Hemophilia A is an X-linked hereditary bleeding disorder that results from either reduced or absent levels of…
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…
Eloctate use in immune tolerance induction (ITI) therapy shows promise in high-risk patients with severe hemophilia A and inhibitors treated for the first time, Bioverativ announced. The results support a potential benefit for some patients who tried and failed ITI with other factors. The study, “Recombinant factor VIII…