Medicines in Development for Rare Diseases 2021 Drug List

An image of a chart of medicines in development for rare diseases 2021

More than 700 medicines are in development targeting many of the approximately 8,000 known rare diseases. Download the full list of medications and therapies for these diseases affecting millions of Americans here.

To read the full report, click here.

An image of a chart of medicines in development for rare diseases 2021