Andrea Lobo, PhD, science writer —

The Spanish Interministerial Commission on the Pricing of Medicines, known as CIPM, has issued a positive recommendation for national reimbursement of Hemgenix (etranacogene dezaparvovec) for eligible people with hemophilia B, according to CSL Behring, the company that markets the gene therapy. The recommendation means that patients in Spain should…

Note: This story was updated Oct. 15, 2024, to note results from the subset of patients with inhibitors participating in BASIS are now expected next year. A European Medicines Agency (EMA) committee has issued a positive opinion recommending the approval of marstacimab in the European Union for…

Having a COVID-19 infection had no significant impact on bleeding episodes, nor on the levels of factor replacement therapy used in treatment, for people with hemophilia, a new study from China found. The study did reveal, however, that, compared with adults, children with hemophilia had a significantly lower…

MGX-001, a gene-editing therapy being developed by Metagenomi for hemophilia A, has shown promising safety and durability in an ongoing preclinical study in three nonhuman primates, the company has announced. Results showed that after one year, two animals had normal (82%) or nearly normal activity levels (41%) of…

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,…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to BE-101, Be Biopharma’s B-cell treatment candidate for hemophilia B. This designation is given to therapies that are meant to treat rare diseases, defined as conditions affecting fewer than 200,000 people in the U.S. It offers…

A European Medicines Agency (EMA) committee has issued a positive opinion recommending conditional marketing authorization of gene therapy Durveqtix (fidanacogene elaparvovec), developed by Pfizer, to treat adults with severe and moderately severe hemophilia B. The European Commission will review the recommendation from the EMA’s Committee for Medicinal…

The U.S. Food and Drug Administration (FDA) has cleared clinical testing in adults of BE-101, a B-cell treatment candidate for hemophilia B being developed by Be Biopharma. The Phase 1/2 trial, called BeCoMe-9, will be a first-in-human study designed to assess the safety and preliminary efficacy of BE-101…

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…

When used long term, Novoeight (turoctocog alfa) safely and effectively prevented and treated bleeding episodes in people with hemophilia A in Japan, according to a real-world study. In fact, Novoeight successfully treated 91.4% of the reported bleeds, and kept bleeding under control in 85.7% of surgical procedures. Moreover,…