FDA Grants Orphan Drug Status for ACE-083 to Treat Muscular Dystrophy

An orphan drug designation is granted by the FDA to help develop safe and effective therapies and treatment options for rare diseases, disorders, or conditions that affect less than 200,000 people in the U.S. Because there are a lot of diseases and disorders that affect a small percentage of people, there are a few drugs and treatment options being studied to treat them. 

This designation is way more than just allowing this drug to be on the market. An orphan drug designation gives pharmaceutical companies grants for clinical trials, as well as a tax advantage. It also allows them to be exclusively on the market for seven years, once the drug receives market approval from the FDA. 

Once orphan drugs are on the market, the market considers them to be just as safe as drugs that are already there. While the orphan drug designation offers significant benefits, they do not allow the drug to bypass the approval process. Orphan drugs have to go through the same amount of clinical trials and research before they are allowed to treat patients.