€1.5m Price Tag Could Spell EU Success For BioMarin’s Hemophilia Gene Therapy
First Hemophilia A Gene Therapy Approval
Executive Summary
The approval is a breakthrough for BioMarin, which is hoping to seize market share from Factor VIII replacement therapies and Roche’s Hemlibra.
You may also be interested in...
CSL/UniQure’s Hemgenix To Enter Uncertain EU Market After Regulatory Nod
The firms’ one-time hemophilia B gene therapy has been approved in the EU just months after a US nod, but concerns remain about its prospects on a market that has seen several setbacks for other gene therapies.
CSL’s Hemgenix Gene Therapy Does Not Need A REMS, FDA Tells National Hemophilia Foundation
Peter Marks says labeling for CSL’s hemophilia B treatment adequately conveys its risks. He also denies NHF request that the label cite eligibility criteria used in clinical trials, responding to a citizen petition on the day the agency approves the therapy.
BioMarin Prepares Investors For A Possible US Hemophilia Gene Therapy Delay
FDA review of the company's BLA for a hemophilia gene therapy could be delayed by three months to allow the agency to review a three-year data analysis from an ongoing trial.