Ultragenyx moved one step closer to establishing a new surrogate biomarker for accelerated approval in rare disease gene therapies. The biotech said Wednesday it will seek accelerated approval for its Sanfilippo syndrome type A gene therapy after the FDA appeared...
The START pilot program aims to accelerate the development of novel drug and biological products for rare diseases rAAV-Olig001-ASPA was selected as one of a few CBER-regulated products based on eligibility criteria, including clinical benefit for rare diseases with...
In a recent discussion, Peter Marks, the director of the FDA’s Center for Biologics Evaluation and Research, and Janet Woodcock, former FDA deputy commissioner, highlighted the critical need for regulatory flexibility in developing rare disease therapies. They...
PARIS and BOSTON, April 22, 2024 – In a significant development in the biopharmaceutical industry, Ipsen and Skyhawk Therapeutics have entered into an exclusive global agreement to advance the discovery and development of novel small molecules that target RNA. These...
Rare diseases currently afflict 300 million people worldwide and 30 million people in the U.S. alone. However, 95% of these diseases lacked an FDA-approved treatment as of January 2023. One reason, industry leaders say, is cost. A 2019 study estimated that orphan drug...
