An FDA advisory committee’s enthusiasm for quality-of-life measures in clinical trials of Morquio A syndrome therapies collided with regulatory reality at the Nov. 19 review of BioMarin Pharmaceutical Inc.’s enzyme replacement therapy Vimizim (elosulfase alfa).
Committee members said quality-of-life (QoL) assessments should be incorporated in future trials of drugs intended to treat Morquio A, also...
Read the full article – start your free trial today!
Join thousands of industry professionals who rely on Pink Sheet for daily insights
- Start your 7-day free trial
- Explore trusted news, analysis, and insights
- Access comprehensive global coverage
- Enjoy instant access – no credit card required
Already a subscriber?