Regulatory agencies in the U.S. and Europe are reviewing applications seeking the approval of fidanacogene elaparvovec, an experimental one-time gene therapy being developed by Pfizer for the treatment of hemophilia B in adults. The U.S. Food and Drug Administration (FDA) expects to complete the review process and…
A researcher at the University at Buffalo has won a three-year grant worth $1.5 million to develop a way to tame a patient’s immune system and prevent it from rejecting factor VIII as a replacement therapy for hemophilia A. “My interest in hemophilia started when I read a mother’s…
Hemophilia treatments in Thailand are limited due to a lack of budgetary resources, the high cost of medications, and an infrastructure inadequate for supporting patient care, a study found. These hurdles mean that people with hemophilia in the Southeast Asian country may not be able to access the care…
Female hemophilia A patients and carriers have more frequent bleeding than males, yet a much smaller proportion receive replacement therapy to prevent or stop them, a U.S. study finds. The medical and pharmacy costs for women with hemophilia A or carriers were up to about 14 times lower…
Xoma closed a $9.6 million deal to buy from Aptevo Therapeutics the rights to receive a payment stream for sales of Ixinity (trenonacog alfa), an approved medication used to prevent and treat bleeding episodes in people with hemophilia B. Under the terms of the deal, Xoma will…
Preventive treatment with Hemlibra (emicizumab) appears to be safe and helps keep bleeding under control in babies who have severe hemophilia A. The number of bleeds was “zeroed out” in nearly half of the babies, and most had zero bleeds requiring treatment. That’s according to interim results from…
SerpinPC, a potential treatment for all types of hemophilia, from the drug development pipeline at Centessa Therapeutics, was found to be safe and to reduce patients’ bleeding episodes for up to 1.5 years. In fact, the therapy candidate has the potential to nearly “zero out” the number of…
The first gene therapy for adults with hemophilia B — branded as Hemgenix (etranacogene dezaparvovec) — has been approved by the U.S. Food and Drug Administration (FDA). Meant to be a one-time treatment, Hemgenix is expected to keep bleeding in check in the long term, while also allowing patients…
Enzyre has raised €12 million (over $12 million) to speed the development of EnzyPad, a device that could one day allow people with hemophilia A to monitor their blood-clotting status in real time from anywhere, including their home. “We are delighted to have closed this successful financing…
Treatment with marstacimab (PF-06741086), an experimental antibody-based therapy developed by Pfizer, reduced the number of bleeds in patients with severe hemophilia who took part in a Phase 1b/2 clinical study. This held true regardless of whether patients had hemophilia A or hemophilia B, or had inhibitors…
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