Hemlibra (emicizumab-kxwh) was recently granted priority review status by the U.S. Food and Drug Administration following the agency’s acceptance of Genentech’s supplemental biologics license application (sBLA) for the treatment for people with hemophilia A without factor VIII inhibitors. Priority review status is granted to therapies that could…

When I was younger, my parents would tell me to date a particular “ideal” man. This man need not be good looking, but he’d better be a good guy — no questionable values, capable of getting along with my family — the perfect guy in the eyes of every Filipino…

The U.S. Food and Drug Administration has approved a larger vial size — 3,500 IU — of CSL Behring’s Idelvion (albutrepenonacog alfa) for the treatment of hemophilia B patients. Idelvion is supplied as a powder for intravenous use and must be reconstituted, or mixed with a liquid (called…

SPK-9001, an investigational gene therapy being developed by Spark Therapeutics and Pfizer, continues to demonstrate great potential to treat hemophilia B patients in an ongoing Phase 1/2 clinical trial. The latest results showed that the annualized bleeding rates of all treated patients were reduced by 98%. In…

Preliminary results of a Phase 1/2a trial of the hemophilia A experimental treatment BIVV001 show a significant extension of replacement therapy lifetime in the blood, Bioverativ announced. The ongoing, and still recruiting, open-label, multicenter EXTEN-A study (NCT03205163) is evaluating the safety, tolerability, and pharmacokinetics of a single intravenous injection…

Should regulatory authorities agree with favorable results from Genentech’s ongoing Phase 3 studies of Hemlibra (emicizumab-kxwh),  hemophilia A patients may have a treatment they can use regardless of inhibitor status, with a choice of three dosing schedules that might better match their lifestyle and preferences. “The ultimate goal for…

Some of my most enjoyable moments are guy times with my boys. We laugh, we attempt to keep the house in order, and we succeed, despite ourselves. My sons, who have my blood running through their veins, remind me that life will be OK; that any problem can be solved,…

Data from ongoing Phase 3 clinical trials show that Genentech’s Hemlibra (emicizumab-kxwh) prophylaxis — or preventive treatment — greatly outperforms current standard-of-care therapies, leading to significant reductions in the number of bleeds in hemophilia A patients regardless of inhibitor status. Promising results from these pivotal studies may lead…

Patient advocacy groups in five states will focus on the challenges affecting patients with blood disorders like hemophilia with the support in part of grants from the Local Empowerment for Advocacy Development (LEAD) program, sponsored by CSL Behring. The five groups are the Bleeding Disorders Foundation of Washington (BDFW), the …

Chronic joint inflammation lowers the health-related quality of life in people with severe hemophilia, a European study reports, and urges physicians to be aware of this association in treating patients. The research, “The impact of severe haemophilia and the presence of target joints on health-related quality-of-life,” appeared in…