Earlier this month, the U.S. Food and Drug Administration gave orphan drug designation to a treatment for a type of liver cancer known as hepatocellular carcinoma (HCC). Called ENMD-2076, the treatment is an oral kinase inhibitor and has previously been approved to treat ovarian cancer, multiple myeloma, and acute myeloid leukemia.
According to the National Institutes of Health, HCC, which is the third deadliest form of cancer, usually occurs in those 50 years of age or older and is caused from cirrhosis in most cases. Cirrhosis is the result of advanced liver disease and scars the organ.
Orphan drug status is reserved for drugs that treat rare diseases that affect fewer than 200,000 people. The Orphan Drug Act of 1983 helped spur development of orphan drugs. The act created financial incentives for drug companies to pursue orphan drug development even though they can only market the drug to a small portion of the population.
In a future issue of Genome, we’ll look in-depth at the state of orphan drugs today as well as the often-high price of medications like these.