EU Approves Hemlibra for Moderate Hemophilia A
The European Commission has approved Hemlibra (emicizumab) as a routine preventive treatment for people with moderate hemophilia A…
Articles by Steve Bryson, PhD
FDA Clears Lab Test Monitoring Hemophilia B via FIX Activity
The U.S. Food and Drug Administration (FDA) has cleared CRYOcheck Chromogenic Factor IX assay, a test intended to be…
FDA Review of Roctavian, Gene Therapy for Hem A, Moving Ahead
A review is continuing into a resubmitted application for approval of Roctavian (valoctocogene roxaparvovec), a gene therapy for adults with…
HEM A and B Study Soon to Open, Leading to Pivotal SerpinPC Trials
Centessa Pharmaceuticals is set to open PRESent-5, an observational feeder study of hemophilia patients who could then…
Canada Approves Rebinyn as a Preventive for Children With Hem B
Health Canada has expanded its approval of Rebinyn (nonacog beta pegol) as a routine preventive treatment for bleeding episodes…
BioMarin Resubmits Request to FDA for Roctavian’s Approval
BioMarin Pharmaceutical has resubmitted an application to the U.S. Food and Drug Administration (FDA) seeking approval of Roctavian…
Untreated Hemophilia A Bleeds Common, Skewing ABR: Analysis
A significant proportion of bleeds in people with hemophilia A are left untreated, according to an analysis of data…
Hem A Gene Therapy Roctavian More Cost-effective Than Hemlibra
If approved for hemophilia A, the one-time gene therapy Roctavian (valoctocogene roxaparvovec) would provide substantial cost savings per patient…
FDA Grants Orphan Drug Status to SerpinPC for Hemophilia B
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to SerpinPC for treating hemophilia B. Orphan…
BioMarin Says Cancer Case in Trial Not Tied to Roctavian Gene Therapy
A participant in a Phase 3 clinical trial evaluating Roctavian (valoctocogene roxaparvovec), a gene therapy for hemophilia A,…