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...
Rocket Pharma is getting ready to light up national landmarks on Thursday as part of its annual rare disease awareness effort, but this year the event is even more meaningful as Rocket awaits an FDA decision on its first drug candidate. Rocket’s gene therapy...
With another quarter exceeding analysts’ expectations, Blueprint Medicines has raised its peak sales estimate for rare disease drug Ayvakit to $2 billion. The Massachusetts biotech unveiled the new target Thursday along with its latest earnings results, which showed...
