UniQure Needs Longer-Term Data For Hemophilia B Gene Therapy EtranaDez
Company Presents HOPE-B Data At R&D Day
Executive Summary
The FDA may want longer follow-up data for companies developing treatments for hemophilia A as well as hemophilia B.
You may also be interested in...
Novartis Can Again Pursue Bigger Zolgensma Market After FDA Lifts OAV-101 Hold
Novartis aims to double the addressable market for its spinal muscular atrophy gene therapy – and catch up to competitors – with the intrathecal version known as OAV-101.
Sigilon Hemophilia Cell Therapy On Clinical Hold, But May Not Be Drug-Related
A patient developed Factor VIII inhibitors, but he had also recently received a COVID-19 vaccine and was genetically predisposed.
Again Please: BioMarin Seeks Speedy EU Review For Hemophilia Gene Therapy
BioMarin is planning to resubmit its EU marketing application for Roctavian soon and, as with the previously pulled application, it wants the regulators to fast-track the new filing.