Small Molecule Oral Delivery For Rare Diseases: Navigating The Complexities As A CDMO

By Rachel Timberlake

Small molecule oral delivery therapies are a crucial part of the treatment landscape for rare diseases, which affect over 30 million people in the U.S.. These low molecular-weight compounds are often preferred by patients for their familiarity, convenience, and potential to aid compliance. They are also more straightforward to manufacture and administer than biologics and allow for dose titration.

However, developing and manufacturing these therapies for limited markets presents significant hurdles. Challenges include high development costs, difficulties in conducting clinical trials with small patient populations, complex small-batch manufacturing, and navigating regulatory pathways.

CDMOs play an indispensable role in overcoming these barriers. They offer specialized expertise, flexible manufacturing for small-scale production, cost-efficient outsourcing, and regulatory support. By leveraging CDMO capabilities, pharmaceutical companies can accelerate development timelines and mitigate risk, ultimately ensuring patients get the treatments they urgently need.