Orphan Drugs & Rare Diseases

Half of rare disease patients are children.

In the United States alone, over 7,000 rare diseases affect more than 30 million people. The FDA has approved hundreds of drugs for rare diseases in the four decades since the Orphan Drug Act was signed into law in 1983. 

To this day, most rare diseases do not have FDA-approved treatments because of the challenges that remain in bringing new medicines for rare diseases to market, ranging from limited R&D budgets to challenging clinical recruitment. 

Our 30+ years of expertise in areas including formulation development, clinical pharmacology, clinical trial drug product manufacturing, and commercial drug product manufacturing make us an ideal partner to help you make a difference in the lives of patients and their families. 

We provide a complete solution to help advance rare disease drug programs:

• Pre-formulation and API characterization
• Formulation development and process development
• First-in-human clinical testing and acceleration into POC trials
• Bespoke clinical trial manufacturing and supply of drug product into Phase I-III patient trials
• Rapid scale-up and commercial drug product manufacturing of low-volume products