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 one of the most common indications, uncomplicated urinary tract infections, while Lilly and Galderma advance biologics for atopic dermatitis

Nearly half of the novel agents approved by the US FDA in the first half of 2024 carried an orphan drug designation

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Recent and Upcoming FDA Advisory Committee Meetings

 
• By Sue Sutter

Recent and upcoming US FDA advisory committee meetings and a summary of the topics covered.

UK Health Data Research Service Looks ‘Encouraging’ For Industry, But Implementation Details Will Be Key

 
• By Anabel Costa-Ferreira

Marcus Vass and Vladimir Murovec of international law firm Osborne Clarke tell the Pink Sheet what the government's planned HDRS might mean for industry, and how it compares with the European Health Data Space.

EU Health Data Space May Speed Up R&D Through Access To Multi-Omics & Clinical Record Data

 
• By Eliza Slawther

The European Health Data Space framework will allow companies to accelerate R&D processes and identify new molecular targets faster by facilitating centralized access to certain types of high-quality data, Finland’s Orion Pharma says.