Rare Disease Rx was founded with one goal in mind: to help you bring orphan drugs to market. Our services include preparing applications for orphan drug designation, providing regulatory strategy, setting up meetings with the Food and Drug Administration (FDA), and more.

There are more than 7,000 rare diseases, defined in the United States as any disease with under 200,000 cases, that affect more than 25 million Americans. Some examples of rare diseases include Tourette's syndrome, cystic fibrosis, Lou Gehrig's disease, and Duchenne muscular dystrophy. 

Benefits of orphan drug designation include marketing exclusivity, federal tax credits, waived fees, qualification for certain research grants, and eligibility to receive guidance from the FDA.

In addition to orphan drug designation applications, we can also help with grant writing, educational content, and more.