Brazilian hemophilia A patients who are 19 or older had significantly higher median care costs per patient per year than younger patients, a study reported. The study also revealed an increase in median costs for patients with more severe disease — that is, those who have less than 1%…

Note: This story was updated July 30, 2024, to clarify patients participating in AFFINE had moderately severe to severe disease, and that a secondary study goal involved the mean treated annualized bleeding rate. Giroctocogene fitelparvovec, an investigational gene therapy for adults with hemophilia A, is generally well…

Novo Nordisk acquired the hemophilia A program and gene-editing technology rights of 2seventy Bio, with 2seventy staffers involved in the program joining Novo Nordisk and continuing to develop the technology to treat a range of diseases other than cancer. 2seventy could receive payments of up to $40…

Loma Linda University Health (LLUH) is now offering the gene therapies Hemgenix (etranacogene dezaparvovec) to people with hemophilia B and SPK-8011 (dirloctocogene samoparvovec), an experimental treatment for hemophilia A. LLUH’s Center of Excellence Hemophilia Program is structured to ensure that financial constraints don’t prevent access to the therapy,…

Note: This article was updated June 26, 2024, to clarify that the analysis of fitusiran in surgery was conducted in patients receiving prophylactic treatment with the therapy. 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…

Efanesoctocog alfa, a long-lasting factor VIII (FVIII) replacement therapy, has now been approved in the European Union for hemophilia A. It will be marketed under the brand name Altuvoct. Altuvoct can be used as an on-demand treatment for bleeding episodes, a once-weekly prophylactic (preventive) therapy to reduce the…

My husband pastors the local First United Methodist Church, where I serve as a deacon. When a parent (or two) goes down the pastoral path, their children are along for the ride. Some preachers’ kids love church, and others, not so much. My youngest son, Caeleb, falls…

A generic version of DDAVP (desmopressin acetate), a therapy approved to control bleeding episodes in people with mild hemophilia A, is available in the U.S. Avenacy planned to begin shipping the therapy the week of April 15, according to a company press release. The generic is available…

Nearly all medical directors at hemophilia treatment centers in the U.S. said they would consider starting preventive Hemlibra (emicizumab-KXWH) in untreated infants with hemophilia A, ages 12 months and younger, a survey study reports. Most respondents also said they’d transition from FVIII replacement therapies, used on-demand to treat…

Hemlibra (emicizumab-KXWH) safely controlled bleeds among hemophilia A patients without inhibitors over nearly five years of preventive treatment, according to long-term data pooled from two Phase 3 clinical trials. “Five years of follow-up data showed a favorable benefit-risk profile of [Hemlibra] prophylaxis,” the researchers wrote in “…