Weekly use of the experimental therapy efanesoctocog alfa maintains therapeutic levels of factor VIII (FVIII) — the missing clotting factor in hemophilia A — and significantly improves physical and joint health, while reducing pain, in adolescents and adults with a severe form of the disease. These are the…

A single dose of the investigational gene therapy BAY 2599023 safely promotes a sustained production of factor VIII (FVIII),  effectively preventing spontaneous bleeds in people with severe hemophilia A, a Phase 1/2 trial shows. Two of the six patients treated to date in this clinical study, both given the…

The protein missing or defective in people with hemophilia A, called blood coagulation factor VIII (FVIII), is able on its own to stimulate immune responses in a mouse model, a study suggests. This immune response to FVIII is dependent on a set of distinct immune cells within the spleen,…

Mim8, an investigational therapy to prevent bleeding episodes in people with hemophilia A, is safe and well-tolerated at multiple doses, according to data from the Phase 1/2 FRONTIER1 trial. The therapy also showed signs of efficacy, with most patients given higher doses experiencing no bleeds during treatment. “We are…

Data from two pivotal Phase 3 clinical trials showed that long-term weekly use of Eloctate can improve bleeding protection while reducing joint symptoms and treatment-associated burden in patients with hemophilia A. Administration of 65 IU/kg of Eloctate per week every 7 days has demonstrated to be sufficient to sustain the required therapeutic effects. These new clinical data further support the prophylactic, or preventive, use of Eloctate, which is currently not indicated for weekly dosing. The findings were the subject of a poster presentation titled “Clinical Outcomes of Weekly Prophylaxis with rFVIIIFc: Longitudinal Analysis of the A-LONG and ASPIRE Study Population” during the 59th ASH Annual Meeting & Exposition that was held in Atlanta, GA. The analysis included clinical data collected from 43 patients older than 12 years, who were treated with weekly dosing of Eloctate for a median time of 3.1 years. All the patients received the treatment during the Phase 3 A-LONG study (NCT01181128) and ASPIRE (NCT01454739) long-term extension study. The data revealed that patients who changed from episodic treatment to weekly prophylaxis with Eloctate experienced a change in median annualized bleeding rates (ABR) of -23.7. In 19 patients who were always on a weekly regimen during the study, the treatment changed their mean ABR from 29 before the study to 1.7 during the trial. This new treatment regimen also provided protection from spontaneous bleeds and joint bleeds, promoting reduced median ABR even when compared to patients who did not receive the weekly regimen. “One of the challenges for people with severe hemophilia A can be treatment every few days with inadequate bleed protection,” Maha Radhakrishnan, MD, senior vice president of medical at Bioverativ, a global biopharmaceutical company that focuses on hemophilia and other rare blood disorders, said in a press release. “We are committed to improving patient outcomes and continue to explore how Eloctate can meaningfully make a difference for patients with the potential for longer dosing intervals that could provide continued joint health improvement.” All participants adhered to the weekly dosing regimen. Indeed, the majority of patients who initiated weekly treatment with Eloctate during the study decided to stay on that regimen. According to the World Federation of Hemophilia guidelines, prophylactic treatment with clotting factor replacement therapy is the better approach to manage severe hemophilia A. However, such treatment commonly requires injections three times per week, which can represent a major burden for the patients. Overall, the data suggests that weekly Eloctate dosing may be an effective prophylaxis regimen for patients with hemophilia A, with improved bleeding protection and reduced treatment burden. “These data show the potential of Eloctate to make a difference for patients, to be able to extend their dosing intervals based on their needs, with improved joint health, and the possibility to reduce the burden of chronic treatment in patients with hemophilia,” said Armin Reininger, MD, PhD, head of medical and scientific affairs at Swedish Orphan Biovitrum (Sobi). Eloctate is a recombinant clotting factor VIII that was engineered based on Fc fusion technology to extend its stability in the body. It is marketed by Bioverativ in the United States, Japan, Canada, and several other countries. The extended half-life recombinant clotting factor is also approved with the brand name Elocta in Europe, where is marketed by Sobi.

Serious bleeds are often a fatal complication in people with hemophilia if not managed early, a 10-year study from Senegal shows. These findings highlight the need for prompt diagnosis of severe bleeding and early administration of replacement therapy to lower the risk of mortality. In resource-limited countries, increasing patients’ and…

Hympavzi (marstacimab-hncq) may be available by prescription in the U.S. before the end of the year. The therapy was approved this month as a routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages 12 and older, with hemophilia A or…

Looking back at my younger self, I don’t think I fully appreciated the help I received from others around me during my school experience. Being one of the few people in school with a rare medical disorder was an interesting experience. It definitely had its perks. For example, school staff…

One-time treatment with the investigational gene therapy SPK-8011 led to sustained low bleed rates for people with hemophilia A in a Phase 1/2 clinical trial. That’s according to data of up to 5 years of follow-up presented by the therapy’s developer, Spark Therapeutics, at the annual meeting of…

Implanting bioscaffolds — 3D biological scaffolds made up of collagen and other molecules — carrying transformed liver cells into rats successfully triggered the production of factor VIII (FVIII), the clotting protein missing in people with hemophilia A. According to researchers, these bioscaffolds may be a therapeutic alternative to FVIII…