The U.S. Food and Drug Administration announced today it approved 21 new orphan drugs to treat rare diseases in
2015, nearly half (47 percent) of all novel new drugs approved for the year. This is the second consecutive year in
which the FDA approved more orphan drugs for rare diseases than any previous year in FDA history, according to
John K. Jenkins, M.D., Director, Office of New Drugs, Center for Drug Evaluation and Research at FDA.
“The remarkable number of orphan drugs approved by FDA in 2015 underscores the progress we are making in
identifying and studying new therapies, the willingness of the investment community to invest in orphan drugs, and
the continued flexibility shown by the FDA in reviewing applications that often include small studies due to the small
number of patients with the disease,” said Peter L. Saltonstall, president and CEO of the National Organization for
Rare Disorders (NORD), the primary independent nonprofit organization representing all patients and families
affected by rare diseases in the U.S. For more details about 2015’s approvals, visit the Novel New Drugs Summary 2015.