2015 was a record-setting year for the U.S. Food and Drug Administration (FDA), as the organization approved 21 new therapies for rare diseases -- more than it ever has in its history.
These therapies, known as orphan drugs, accounted for 47 percent of all novel drugs approved by the FDA in 2015, Dr. John Jenkins, director of the FDA's Office of New Drugs, Center for Drug Evaluation and Research, said.
“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,” Peter Saltonstall, president and CEO of the National Organization for Rare Disorders (NORD), said. “At the same time, we must recognize that our patient community faces new challenges in accessing many new therapies, and that the vast majority of the 7,000 identified rare diseases still have no treatment options.”
Saltonstall would like to see the FDA's trend of improving record numbers of orphan drugs continue.
“We look forward to still more approvals in 2016 and beyond,” he said.
