Centessa discontinues development of SerpinPC for hemophilia
Nov 15, 2024 · The company was also conducting a long-term open-label extension study called PRESent-6 (NCT06568302) to evaluate the safety and efficacy of SerpinPC for up to 25 months in patients who had completed other trials of the therapy.. SerpinPC was granted fast track and orphan drug status by the U.S. Food and Drug Administration for hemophilia B. The designations are intended to speed the ...
IND-enabling studies for hemophilia A gene-editing therapy to …
Nov 19, 2024 · This approach should lead to a sustained increase in FVIII activity, normalizing blood clotting and preventing bleeds with hemophilia A. In a preclinical study, three nonhuman primates were treated with a single dose into their veins, or intravenously, of the AAV containing the F8 gene, followed by a single intravenous dose of the nanoparticles a month later.
FDA approves marstacimab, now Hympavzi, for hemophilia
Oct 11, 2024 · Hympavzi approved for adults and children, ages 12 and older. Hemophilia is a rare disease caused by a missing or faulty clotting factor — a protein that helps the blood to clot and prevents the condition’s hallmark symptom, excessive bleeding.In hemophilia A, the missing or dysfunctional clotting factor is factor VIII, or FVIII, while in …
Hemophilia A, B prophylaxis therapy Hympavzi may soon be …
Oct 25, 2024 · Developer Pfizer has tagged Hympavzi with a wholesale acquisition cost of $795,600 per year, which compares to the “pricing for prophylactic treatments for hemophilia A or B now on the market,” Younos Abdulsattar, Pfizer’s U.S. rare disease medical lead, said in a Q&A with Hemophilia News Today.. Patients’ actual out-of-pocket costs will vary based on their insurance coverage and many ...
Concizumab reduces bleeding in patients without inhibitors
Nov 29, 2024 · That’s according to data from explorer8 (NCT04082429), an ongoing Phase 3 clinical study in which Novo Nordisk’s concizumab, delivered daily under the skin for at least 32 weeks (seven months) using an injector pen reduced mean annualized bleeding rates by 86% in patients with hemophilia A and by 79% in those with hemophilia B.. The data are consistent with that from explorer7 (NCT04083781 ...
Hemophilia bypassing agent Sevenfact lowers bleeds in patients...
Oct 18, 2024 · The bypassing agent also was administered where patients showed a poor response to NovoSeven RT (eptacog alfa), an approved bypassing agent marketed by Novo Nordisk.. Sevenfact was effective in 96% of the cases at controlling bleeds and resolving symptoms when used as a first-line therapy, results showed.
Committee recommends EU approval of concizumab for hemophilia
Oct 22, 2024 · However, an application for concizumab’s approval in the U.S. was rejected by the U.S. Food and Drug Administration early last year, with requests for more information on the monitoring of patient dosing and the treatment’s manufacturing process. Novo Nordisk has already refiled its application seeking the therapy’s approval in the U.S., according to an earnings call hosted by the ...
Hemophilia gene therapy Beqvez reduces bleeds in Phase 3 trial
Oct 8, 2024 · That’s according to final data from BENEGENE-2 (NCT03861273), the Phase 3 study that supported this year’s approval of Beqvez as a one-time treatment for adults with moderate to severe hemophilia B in the U.S. Beqvez is also approved in Canada and the European Union to treat moderately severe to severe hemophilia B. “What we saw from patients in this study was that within a few days of ...
Roctavian effective for hemophilia A over 7 years, per trial update
Feb 7, 2024 · That’s according to an update on a Phase 1/2 clinical trial (NCT02576795), launched in the U.K. in 2015, that’s testing the safety and efficacy of increasing doses of the gene therapy, which was approved in the U.S. in June 2023.. The new data is being presented by BioMarin Pharmaceutical, Roctavian’s developer, at the 2024 European Association for Haemophilia and Allied Disorders (EAHAD ...
Hemophilia A vs. B: Differences and similarities - Hemophilia News …
Dec 1, 2023 · Hemophilia A is more common. It accounts for approximately 80% of all hemophilia cases. Hemophilia B is the second most prevalent type of the bleeding disorder, making up the remaining 15% to 20% of cases.. Though they have different causes, hemophilia types A and B do also share similarities, with both occurring primarily in …