A single dose of Spark Therapeutics’ investigational gene therapy SPK-8011 increased the levels of factor VIII (FVIII) — the missing clotting protein in hemophilia A — and, in 16 of 18 male patients, those levels were sustained for up to four years, according to results from a Phase 1/2 clinical…
A partnership between Emmes and MedGenome seeks to accelerate development of genomics-powered therapies for people with rare diseases, such as hemophilia. The companies plan to combine patient data into custom disease registries to improve research design and help with patient recruitment for clinical trials. In addition, they…
Canadian Blood Services (CBS) has awarded a three-year tender — an exclusive bulk purchase agreement aiming to minimize medication cost — to Takeda Canada for the purchase of Adynovate, an injectable replacement therapy for the prevention and treatment of bleeding episodes in people with hemophilia A. This…
In an unexpected turn, Catalyst Biosciences is discontinuing the clinical development of marzeptacog alfa activated (MarzAA), its experimental under-the-skin therapy for hemophilia A and B patients with inhibitors, which was being evaluated in an international Phase 3 clinical trial. “Based on several factors including a recently updated feasibility assessment,…
When given monthly to people with hemophilia A or B with inhibitors, fitusiran significantly reduced bleeding, and for some, fully eliminated them, according to data from the Phase 3 ATLAS-INH study.
Chugai Pharmaceutical is asking regulatory authorities in Japan to extend the use of Hemlibra (emicizumab) in preventing or reducing the frequency of bleeding episodes to people with acquired hemophilia A. Hemophilia A is caused by genetic mutations that make the body unable to produce a fully functional…
The U.S. Food and Drug Administration (FDA) has placed a clinical hold on the Phase 3 trial evaluating SB-525 (giroctocogene fitelparvovec), an investigational gene therapy for hemophilia A. This pause in study recruitment and dosing was taken to give the agency time to review changes to the AFFINE…
Jim Christensen Jr., a hemophilia A patient who has supported the National Hemophilia Foundation (NHF) for more than three decades, has received the Lester A. Rosen Humanitarian and Achievement Award, given annually by Ameritas, for which he is an independent financial representative. With the award, the insurance company…
Chronic pain and a reliance on pain killers remains high among adult men with severe hemophilia B after two years on preventive treatment with extended half-life recombinant factor IX — even with a lesser bleeding frequency and fewer repeated joint bleeds, a real-world study from Ireland reports. These findings…
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to GENV-HEM, GeneVentiv Therapeutics’ investigational gene therapy for hemophilia A and B patients, with or without clotting factor inhibitors. Orphan drug designation is given to treatment candidates that have the potential to be safe and effective…
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