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Related: Editorials & Other Articles, Issue Forums, Alliance Forums, Region ForumsPatient Dies After Taking Pfizer's Drug for Blood Disorder
The death occurred on Dec. 14 after the patient experienced serious adverse events, including a stroke and brain bleeding, according to a statement from the European Haemophilia Consortium, a patient group. The patient was participating in an open-label extension study of Hympavzi, which is approved to treat the condition that prevents blood from clotting properly and can lead to life-threatening bleeding in muscles, joints and organs.
Last year, regulators in the US and Europe approved Hympavzi to prevent or reduce the frequency of bleeding episodes in certain patients with hemophilia. While other treatments exist, Pfizers drug was billed as the first of its kind. Instead of replacing clotting factor, it targets a protein in the blood clotting process.
In a statement, Pfizer said its gathering more information to better understand the circumstances surrounding the patients death. The company said it doesnt expect any impact to safety for patients treated with the drug, citing current knowledge and the overall clinical data collected to date.
https://www.bloomberg.com/news/articles/2025-12-23/patient-dies-after-taking-pfizer-s-drug-for-blood-disorder
haele
(15,030 posts)Poor patient.
Genetic based medication is amazing, and could be a lifesaver in 99.9% of the treatment, but that one patient did not have the genetics that could tolerate the medication, so it has to be put on hold until they can find out what caused that reaction and see if they need to test for that genetic condition before they continue with the treatment.
hlthe2b
(112,627 posts)Treating these blood diastheses (clotting disorders, whether from hemophilia or autoimmune, sepsis, or severe traumatic conditions) is such a daunting proposition that most cannot appreciate. They (and the media) will see that a patient receiving the experimental treatment died and while rightfully horrified, will not find the necessary context to appreciate how serious the condition is, how deadly, and how desperately we need effective drugs to treat it. I hope this patient's death will provide the information that might help refine this and other drugs or the recommendations that might ultimately target their use, but likewise that it will not halt cautious testing and research--even on this drug.