Catalyst Biosciences recently announced updates on two of its lead compounds — Factor VIIa (FVIIa) marzeptacog alfa (activated) (MarzAA) and Factor IX (FIX) dalcinonacog alfa (DalcA) — for treatment of hemophilia A or B with inhibitors, and for hemophilia B. DalcA is a potent recombinant (artificial) Factor IX prophylactic therapy designed to prevent acute bleeding…

Oral hygiene among children and adults with severe hemophilia is poorer than in healthy controls, a single-center study suggests. The study, “Oral hygiene and dentition status in children and adults with hemophilia: A case–control study,” was published in the journal Special Care Dentistry. Dental care is a…

Prophylaxis with Idelvion (rIX‐FP) significantly improved clinical outcomes and quality of life for children with hemophilia B and eased daily life for their caregivers, Phase 3 clinical trial data show. The findings of the study, “Health-related quality of life in paediatric haemophilia B patients treated with…

Treatment of severe hemophilia A with Eloctate and hemophilia B with Alprolix was safe and improved the patients’ annualized bleed rates (ABRs) over four years, according to the results of two extension studies announced by Bioverativ and Sobi. The two open-label extension studies — ASPIRE (NCT01454739), and B-YOND (NCT01425723) — evaluated…

Dear hemophilia, Truth be told, I hate you sometimes. I especially hate you when you suddenly pop out of nowhere and find ways to ruin my husband’s life. I hate you when you encroach on plans made weeks in advance; long-standing plans that were carefully and painstakingly thought out. As…

A new analysis of the Phase 3 HAVEN 2 study’s results shows that Genentech’s Hemlibra (emicizumab-kxwh) prophylaxis (preventive treatment) leads to significant reductions in the number of bleeds among hemophilia A children younger than 12 with factor VIII inhibitors. The results showed that Hemlibra administered on different schedules — once a…

Health Canada recently authorized Shire Pharma Canada‘s treatment Adynovate for hemophilia A patients younger than 12 based on results of a Phase 3 clinical trial. Approved Nov. 21, the injectible factor replacement therapy is available to pediatric patients for the prevention and control of bleeding episodes, as well…

Jivi (BAY94-9027), a lab-made version of the blood clotting factor VIII (FVIII), has been approved by the European Commission for the prophylactic (preventative) treatment of hemophilia A in patients older than 12, Bayer announced. The recommended prophylaxis for Jivi administration is every five days, every…

Gene therapy has the potential to stop hemophilia in its tracks. But such therapy will likely be costly, and can carry risks that have generated some apprehension among patients who could benefit most from it. That’s the message from hemophilia patient advocates who spoke at the 2018 NORD Rare Diseases…

A gene therapy from uniQure in early testing, called AMT-180, has the the potential to treat all hemophilia A patients, including those with inhibitors, according to the company. Hemophilia A is caused by missing or defective factor VIII (FVIII), a clotting protein. About 30% of patients with severe hemophilia A develop inhibitors, or…