In the fall of last year Genentech reported that during a routine examination of batches of Hemlibra®, they found translucent particles composed of a therapeutic protein and an organic, non-toxic silicone oil (polydimethylsiloxane). While it is not…
The National Hemophilia Foundation (NHF) and Hemophilia Federation of America (HFA) continue to follow up on issues arising from the recent recall of Stimate (desmopressin) nasal spray, which is manufactured by Ferring Pharmaceuticals and…
The National Hemophilia Foundation’s (NHF) Medical and Scientific Advisory Council (MASAC) issued two new documents, which were adopted by NHF’s Board of Directors on September 3, 2020.
MASAC Document #262 addresses drawbacks…
Last month, BioMarin learned that approval for their investigational hemophilia A gene therapy was not forthcoming but would in fact be delayed for some time, pending additional safety and efficacy trial data. While the company…
Objective:
Kids B-LONG was an open-label phase 3 study that evaluated the safety, efficacy, and pharmacokinetics (PK) of rFIXFc in previously treated children (aged <12 years; ≥50 prior exposure days [EDs] to FIX) with severe…
On September 1, 2020, NHF and HFA learned that Mylan N.V. is conducting a voluntary nationwide recall of four lots of Tranexamic Acid Injection, USP 1000 mg/10 mL, packaged in cartons of 10 single-dose 10 mL vials. Tranexamic acid…
Mylan N.V. today announced that its U.S.-based Mylan Institutional LLC business is conducting a voluntary nationwide recall to the hospital/clinic level of four lots of Amiodarone HCl Injection, USP 450 mg/9 mL, packaged in cartons of 10 single-dose…
What comes to mind when you think about the future? How does living with an inherited bleeding disorder, or caring for someone who does, look different for the next generation? What do you want to see coming from research done by leaders across the…
The National Hemophilia Foundation (NHF) has received a five-year cooperative agreement from the Centers from Disease Control and Prevention (CDC). The collaboration, which gives NHF $500,000 a year for five years, will be used to support…
Biomarin announced that they received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) on August 18th for their investigational hemophilia A gene therapy valoctocogene roxaparvovec. The CRL indicates that while the…