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...
Keeping Track: US FDA Receives Advanced Biologic BLAs From ImmunityBio, CSL; Nymox, Medac Stumble
The latest NDA and BLA submission news and highlights from the Pink Sheet’s US FDA Performance Tracker
uniQure/CSL’s EtranaDez Could Take Lead Position In Hemophilia B Gene Therapy Market
The therapy could also face competition from players like Freeline, but new Phase III data position uniQure and CSL Behring well for planned FDA and EMA filings.
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.