The purpose of the study is to evaluate the efficacy and safety of this investigational gene therapy in patients with moderately severe to severe hemophilia A.
CSL Behring provided a statement to NHF and HFA regarding the recall of Stimate® (desmopressin) nasal spray.
BIVV001 is a novel, investigational recombinant factor VIII therapy developed for the prevention of bleeding episodes in hemophilia A patients via once weekly prophylactic infusions.
The Hemophilia Alliance joined with NHF and HFA requesting additional information from Ferring Pharmaceuticals and CSL Behring around the recent Stimate recall.
Study investigators will be looking at how trial participants may develop antibodies against the AAVs that are a key component to Biomarin's gene therapy.
Tranexamic acid injection is indicated in patients with hemophilia for short term use to reduce or prevent hemorrhage and reduce the need for replacement therapy during and following tooth extraction.
BioMarin plans to meet with the FDA in the coming weeks to align on the next steps to obtain approval.
Concizumab is an investigational therapy designed to be equally effective in individuals with hemophilia A and B, irrespective of inhibitor status
The recall has been expanded from the pharmacy level to the patient, or consumer level.
CSL Behring and Ferring Pharmaceuticals, Inc have responded to the joint NHF-NHF letter of July 22, 2020, regarding a recent recall of Stimate (desmopressin) nasal spray.
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