News

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…

Finding treatments and potential cures for rare diseases is crucial, but so is the quality of patients’ lives — a rather nebulous term that means different things to different people. “Recently, there’s been much more of a focus on Quality of Life (QoL) issues, real-world evidence and patient-reported outcomes,” said…

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…

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…

The National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both the 1983 Orphan Drug Act and NORD’s founding at a dinner tonight in Washington, D.C. The 2018 Rare Impact Awards, to be held at the Andrew W. Mellon Auditorium, will be webcast via Facebook for those…

The Arizona Hemophilia Association (AHA) is inviting camp professionals in the bleeding disorders and hemophilia communities to participate in the National Camp Exchange (NCE) program. Applications are open until May 30 for 2018 summer programs. NCE is set up to give those who plan, organize, and operate…