Genentech will present more than 60 abstracts at the 58th American Society of Hematology Annual Meeting (ASH 2016). Nine of Genentech’s approved or investigational medicines will be featured, including Gazyva (obinutuzumab), Rituxan (rituximab), Venclexta (venetoclax) and emicizumab, the company’s investigational drug in hemophilia. More specifically, Genentech will present…
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Men with moderate-to-severe hemophilia A are again being enrolled in the Phase 3 AFFINE trial, which is testing the safety and effectiveness of SB-525 (giroctocogene fitelparvovec), an experimental gene therapy being developed by Pfizer and Sangamo Therapeutics. Pfizer, the study’s sponsor, had placed a voluntary pause…
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),…
I entered the bedroom of my youngest son, Caeleb, one recent morning to offer him my usual cheerful greeting. That’s when I found him lying in bed in excruciating pain. Caeleb has hemophilia A, and years of damage from internal bleeding episodes have left him with little…
Four people in a region of Northern Italy were diagnosed with acquired hemophilia A (AHA) weeks after receiving the second dose of a vaccine against COVID-19, scientists reported. The unusually high number of newly identified acquired hemophilia A cases within eight months in the Italian province led the investigators to suspect…
Therapeutic plasma exchange (TPE) was found to reduce bleeding and ease disease symptoms in six people with acquired hemophilia A (AHA), a case series reported. These findings support TPE — in which the liquid part of blood is replaced with substitute plasma — as an alternative treatment option in…
My wife, Cazandra, and I enjoy speaking on topics critical to the bleeding disorders community, as both of our adult sons live with hemophilia. A few weeks ago, we had the chance to lead a seminar called “Telling Our Stories” at the National Bleeding Disorders Foundation’s (NBDF) annual…
A single dose of Roctavian (valoctocogene roxaparvovec-rvox), a gene therapy for severe hemophilia A from BioMarin Pharmaceutical, maintains factor VIII (FVIII) levels at near-normal or normal levels for up to five years, with most patients staying off prophylaxis, or preventive treatment. These lasting benefits were observed in GENEr8-1…
Researchers investigated self-reported health-related quality of life (HrQoL) in children and adolescents with hereditary bleeding disorders, such as hemophilia A, and found no differences between patients and their healthy siblings and peers. The research paper, titled “Health-Related Quality of Life in Children and Adolescents with Hereditary Bleeding Disorders…
One year of treatment with Elocta (efmoroctocog alfa) — an extended half-life replacement therapy known as Eloctate in the U.S. — reduced the frequency of bleeds in the elbows and knees of adults with severe hemophilia A, a Spanish study showed. Reduced pain intensity in the elbows also…