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 …
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 ...
Bone mineral density low in hemophilia patients: Study
Dec 6, 2024 · Bone mineral density, or the amount of mineral in bone tissue, is decreased in 63.1% of people with hemophilia, according to a study from Germany. Study findings also indicated the degree of loss depended on hemophilia severity. Patients with severe hemophilia had lower bone mineral density in the ...
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 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 ...
Prophylaxis is able to reduce bleeds from severe hemophilia in...
Sep 27, 2024 · Annualized bleed rate reduced. Researchers analyzed four clinical trials involving patients with severe hemophilia A and B, ages 50 and older, who received treatment with extended half-life products to better understand the impact of prophylaxis when introduced later in life.. The hemophilia A patients had taken part in A-LONG (NCT01181128) and its ASPIRE (NCT01454739) extension study, while ...
Marstacimab recommended for EU approval for hemophilia A and B
Sep 24, 2024 · A European Medicines Agency (EMA) committee has issued a positive opinion recommending the approval of marstacimab in the European Union for preventing bleeding episodes in people with severe hemophilia A or hemophilia B without inhibitors.. The European Commission will now review the recommendation, made by the EMA’s Committee for Medicinal Products for Human Use (CHMP), with a final ...
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 is reviewing fitusiran application for hemophilia A, B, with...
Jun 26, 2024 · The U.S. Food and Drug Administration (FDA) is reviewing a request to approve fitusiran, an under-the-skin injection therapy, as a treatment for adults and adolescents with hemophilia A or hemophilia B, with or without inhibitors.. A decision is expected by March 28, 2025, according to fitusiran’s developer Sanofi. “We look forward to working in partnership with regulatory agencies to keep ...
13 Fast Facts About Hemophilia – Hemophilia News Today
Jun 15, 2017 · Here are a few fast facts about this rare blood disease: MORE: How hemophilia is inherited. There are three different types of hemophilia: hemophilia A, B, and C. The type is determined by the clotting factor the person is deficient in.