Hemophilia Archives - MPR

Hemophilia

MPR Weekly Dose Podcast Episode 48

Treatment shows promise for patients who had pneumonia associated with COVID-19; A COVID-19 test that may have given inaccurate results; Neck gaiters as face masks may actually spread the virus; Drug applications are halted by the FDA; And Narcan has it’s shelf life extended. Also, we recap drug pipeline news from the past week.

BLA Submitted for Gene Therapy to Treat Hemophilia A

BioMarin has submitted a Biologics License Application (BLA) to the Food and Drug Administration (FDA) for valoctocogene roxaparvovec (BMN 270) for the treatment of hemophilia A in adults. This is the first marketing application submission for a gene therapy product for any type of hemophilia. Valoctocogene roxaparvovec is an investigational adeno-associated virus (AAV) gene therapy…

Investigational Therapy for Hemophilia A Granted Orphan Drug Designation

The Food and Drug Administration (FDA) has granted Orphan Drug designation to SIG-001 (Sigilon Therapeutics) for the treatment of hemophilia A. SIG-001 utilizes Sigilon’s Shielded Living Therapeutics platform to implant engineered human cells to produce stable blood plasma levels of factor VIII. The cells are also shielded by Sigilon’s proprietary Afibromer biomaterials matrix that minimizes…

Esperoct Approval Provides New Treatment Option for Hemophilia A

Novo Nordisk announced that the Food and Drug Administration (FDA) has approved Esperoct (turoctocog alfa pegol, N8-GP) for the treatment of patients with hemophilia A (congenital factor VIII deficiency) for routine prophylaxis to reduce the frequency of bleeding episodes, on-demand treatment and control of bleeding episodes and perioperative management of bleeding.