News & Analysis on the Clinical Development and Manufacture of Large Molecule Drugs
Hot on the heels of last year’s EU approval of Hemgenix, the first gene therapy for the blood disorder hemophilia B, the UK and Denmark are the latest European nations to take steps to pay for the treatment.
The US FDA has delayed approval of Rocket Pharmaceuticals’ gene therapy for treatment of a rare immune condition known as leukocyte adhesion deficiency, citing a need for more information about the manufacturing process.
Results from the Vivacity-MG3 study will support Johnson & Johnson’s application for regulatory approval of nipocalimab.