There’s a good chance those who don’t have a close connection with hemophilia don’t know much about the disease. Some may know that it’s a bleeding disorder, though they probably don’t know what the outcomes are for those who are diagnosed or how it’s treated. It’s for these…

First in a two-part series. I had never heard of hemophilia until the doctors came into my hospital room the day after my first son, Julian, was born. I soon learned that about two-thirds of those diagnosed with hemophilia are because of a spontaneous mutation. Simply…

Among its corporate goals for 2018 announced recently, Alnylam Pharmaceuticals said it will continue to clinically develop the investigational RNA therapy fitusiran to treat hemophilia. Fitusiran, also known as ALN-AT3SC, is an RNAi therapeutic agent that targets a protein called antithrombin — which inhibits blood clotting — while increasing production of the…

When you have a year that is best left in the past, ushering in the new year is a time of celebration. You start with a clean slate and an attitude of hopefulness for what is yet to come. For many years I based the quality of a…

The U.S. Food and Drug Administration (FDA) has lifted the hold on clinical studies with fitusiran, an investigational therapy for hemophilia, Alnylam Pharmaceuticals announced. “We are pleased with the FDA’s decision to lift the clinical hold, as fitusiran holds the potential to help improve the lives of people…

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.

Injection of Hemlibra (emicizumab-KXWH) every four weeks helps control bleeding in hemophilia A patients, an interim analysis of the Phase 3 HAVEN 4 study shows. The results, announced by Genentech, are in agreement with several previous clinical trials – HAVEN 1 (NCT02622321),…

Buckets. Buckets and buckets of tears. That’s the only way I can describe the tears I have shed over the years because of living with hemophilia in my home. Both of my sons have had their own struggles with hemophilia and inhibitors, and the tears I have shed…

The U.S. Food and Drug Administration (FDA) recently granted orphan drug status to rofecoxib (TRM-201), an non-opioid investigational pain treatment for hemophilic arthropathy (HA, a hemophilia degenerative joint disease) under development by Tremeau Pharmaceuticals. Rofecoxib is a COX-2 selective non-steroidal anti-inflammatory drug (NSAID) with a well-established efficacy profile. It is…

Hemophilia in children is diagnosed at an average age of 9 months, and most children are diagnosed by their second birthday. If your pediatrician suspects your child has hemophilia, you may be concerned about their first appointment with the hematologist. To ease your worries, we’ve put together some tips to help you through…