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…

Last Friday, my youngest son, Caeleb, and I started our day as usual and ate at one of our favorite restaurants, Barelas Coffee House in Albuquerque, New Mexico. Summer carries a special magic in the MacDonald home. For many years, I’ve enjoyed spending Museum Fridays with my sons,…

One night when my oldest son, Julian, was 6, we sat down at the kitchen table, preparing to infuse factor VIII, a clotting agent, to treat his hemophilia. He held out his arm, and I looked at the vein we called “Old Faithful.” Located on the top of…

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…

To help advance new treatments for hemophilia and other blood disorders, Pathway to Cures (P2C) has invested $250,000 in Anvesana, a company working to develop therapies based on RNA biology. “Anvesana is delighted to be working with Pathway to Cures to develop therapeutics that will bring life altering…

I am originally from Texas, so I know the meaning of the word “team.” The Lone Star State reveres sports organizations like no other place in the country. Friday nights in the fall are overwhelmed with football, marching bands, and great times to visit with friends. Everyone gathers together with…

One recent evening, my wife and I watched our favorite television shows. After the last one ended, the local news came on to give us the latest updates on the day’s top stories. One segment touched me deeply, and I couldn’t help but think back on the many health difficulties…

Raising awareness is a delicate task. Sometimes, the most effective way to promote dialogue is with a tool that is considered controversial. I understood this seven months ago when I made a T-shirt that was bound to attract both people who loved it and people who hated it.

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…