On May 1, 2012, the U.S. Food and Drug Administration (“FDA”) approved a new orphan drug to treat Gaucher disease. The drug, Elelyso (taliglucerase alfa), is an injection therapy used for enzyme replacement in people with the rare genetic disorder. Gaucher disease is essentially an enzyme deficiency that causes the buildup of lipids in several organs, including the liver and kidneys. Approved specifically to replace the missing enzyme (glucocerebrosidase) in patients with a confirmed diagnosis of Type 1 Gaucher disease, the drug underwent testing and approval through the FDA’s orphan drug designation process.
Pursuant to the Orphan Drug Act of 1983, the FDA is responsible for the development and implementation of the orphan designation process. Through this process, drug sponsors are able to more easily obtain approval, while receiving certain tax benefits. Because orphan products are meant to treat those with very rare illnesses, these incentives were put in place to prompt drug and device sponsors in developing products that otherwise would be unprofitable to pursue. To illustrate how rare such a condition must be in order to qualify for orphan designation, Type 1 Gaucher disease is believed to afflict approximately 6,000 people in the United States. In order to obtain orphan drug status, FDA regulations require that the disease or condition for which the drug is intended to treat affects fewer than 200,000 persons per year in the United States. 21 C.F.R. part 316. For more information about obtaining an orphan designation, please contact us at firstname.lastname@example.org.