FDA Updates Label of Bioverativ’s Alprolix Upon Review of Positive Interim B-YOND Trial Data
The U.S. Food and Drug Administration (FDA) has approved an update to the label of Alprolix, based on long-term…
Articles by Alice Melão
HAVEN 3 Data Supports Use of Hemlibra to Treat Hemophilia A Patients Without Factor VIII Inhibitors
Administration of once-weekly Hemlibra (emicizumab-kxwh) significantly reduced bleeding rates in patients with Hemophilia A compared to other clotting…
New Version of Possible Gene Therapy for Hemophilia B, SPK-9001, Enrolling More Patients in Trial
An ongoing Phase 1/2 clinical trial evaluating a gene therapy, SPK-9001, in people with hemophilia B is expanding its enrollment…
BioMarin’s Investigative Gene Therapy for Hemophilia A Wins FDA Breakthrough Designation
The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to BioMarin Pharmaceutical’s investigative therapy, valoctocogene roxaparvovec to…
FDA Grants Investigational New Drug Status for BioMarin’s BMN 270 Hemophilia A Therapy
The U.S. Food and Drug Administration (FDA) has approved BioMarin Pharmaceutical‘s…
Economic Burden of Prophylaxis is Surpassed by Long-Term Benefits for Hemophilia B Patients, Analysis Shows
Researchers have found that the high price of recombinant clotting factors for patients with hemophilia B is the main…
Researchers Offer a Rundown on New Approaches to Treating Hemophilia
Hemophilia patients are taking heart from a number of innovative treatment approaches that have reached the clinical trials stage. Researchers…
Improved Medicines, Tailored Strategies May Improve Hemophilia Treatment Adherence, Study Finds
Knowledge about the clinical benefits of treatments and their importance in the outcome of patients, as well as more stable…
PRO Instruments Offer Reliable Data to Assess Hemophilia Patients, Study Shows
U.S. hemophiliacs who use patient-reported outcome (PRO) instruments can provide reliable information on their disease burden, finds a new study…
ICER Draft Report on Emicizumab as Potential Hemophilia A Therapy Now Open to Public Review
The Boston-based Institute for Clinical and Economic Review (ICER) says a “draft scoping document” outlining its planned comparative review of…