1. Pink Sheet

Rare Diseases Rule, But Some Common Conditions Stand Out In Pending US Applications

 
• By Bridget Silverman

Half of the FDA’s upcoming 2024 user fee goals target rare diseases, but highly prevalent conditions like uncomplicated urinary tract infections and atopic dermatitis are also up for agency action in the second half of the year.

Stones in the balance
The FDA’s upcoming decisions will address many rare diseases and a few common ones. • Source: Shutterstock

Rare diseases are almost certain to remain one of the biggest areas for novel drug development in 2024, but the orphan drug pipeline will be making space for some novel therapies for common conditions over the next six months.

Key Takeaways
  • FDA is on track for a fourth consecutive year of rare disease therapies making up 50% or more of novel drug approvals.
  • Iterum’s oral sulopenem is seeking...

Nearly half of the novel agents approved by the US FDA in the first half of 2024 carried an orphan drug designation. The second half looks similar, with more than...

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