When I talk to most people outside of the medical/pharma world, they have never heard of an orphan drug. The description brings a queer smile to their face, as they image some sort of little orphan Annie drug. However, unbenounced to most people, orphan drugs are likely future of the drug industry.

To begin, let us define exactly what an orphan drug is. The FDA states that an orphan drug is a product used for “the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S.” With over 317 million people in the U.S., a drug that affects less than 200 thousand seems insignificant, but looks can be deceiving. The U.S. population will start to see these drugs on their shelves in the next two decades via two ways.

The first is the use of drugs, initially approved for orphan status, repurposed to treat common disorders. Does this seem an unlikely drug discovery strategy? Actually common disorders have benefitted from rare genetic disorder research for a long time. For instance, Familial Hypercholesterolemia (FH) is a rare genetic disease characterized by high “bad cholesterol” levels. Studies of FH patients in the 1970s and 80s informed the discovery of statins (e.g. Lipitor), one of the most widely used class of drugs on the market.

Today, companies like Zafgen are taking advantage of orphan drug status to get approval of compounds they hope will treat the greater population. Zafgen’s beloranib treats obesity in individuals with Prader-Willi syndrome, a rare genetic disorder caused by a partial deletion of chromosome 15. Beloranib targets an important metabolic enzyme deficient in individuals with Prader-Willi syndrome and likely a subsection of individuals with severe obesity in the greater population. Therefore, beloranib’s approval by the FDA may bring a new drug to market with the potential of treating obesity in the common populace.

The second way orphan drugs will become a greater consideration is through personalized medicine. As genomics takes on a larger role in medical decisions, the ‘one drug, one disease’ model will no longer be relevant. Drugs will progress towards specific genetic populations, as revealed through an individual’s particular genetic background. So next time you hear the term ‘orphan drug’ keep an eye out, your medicine cabinet may be giving it a home very soon.