How Often are Orphan Drugs Orphaned by the Thermochemical Community?

Kathleen F Edwards; Joel F Liebman

doi:10.2174/0929867325666181101120058

How Often are Orphan Drugs Orphaned by the Thermochemical Community?

Curr Med Chem. 2020;27(1):23-31. doi: 10.2174/0929867325666181101120058.

Authors

Kathleen F Edwards¹, Joel F Liebman^{1

2}

Affiliations

¹ Doctor of Business Administration Program, The Graduate School, University of Maryland Global Campus, Largo, MD 20774, United States.
² Department of Chemistry and Biochemistry, University of Maryland, Baltimore County, Baltimore, MD 21250, United States.

PMID: 30381059
DOI: 10.2174/0929867325666181101120058

Abstract

Orphan drug products (e.g. drugs and biologics) in the United States are those that treat people with rare chronic diseases, often cancer or metabolic disease. The rare disease condition being treated by these orphan drugs must serve a patient population of less than 200,000 people in the U.S. in order to earn the orphan drug product title. Just as the disease conditions are seen as "orphans," so, we assert is the thermochemical understanding of the drugs themselves in terms of the chemical structures that define those drugs. This article illustrates this orphan thermochemical status for a recent series of orphan drugs.

Keywords: Orphan drug products; fabrazyme; metabolic disease; orfadin; thermo-chemical drugs; thermochemical..

MeSH terms

Drug Approval
Orphan Drug Production*
Rare Diseases
United States
United States Food and Drug Administration