Nearly Half of New Drugs Approved in 2016 Were Orphan Drugs

January 17, 2017

Among the 9 new orphan drugs approved by the FDA in 2016 were 3 treatments for rare diseases that, so far, had no approved treatments: Duchenne muscular dystrophy, spinal muscular atrophy, and severe hepatic veno-occlusive disease.

Close to half (41%) of the new medications approved by the FDA in 2016 were orphan drugs, according to the National Organization for Rare Diseases (NORD). Among the 9 new orphan drugs were 3 treatments for rare diseases that, so far, had no approved treatments: Duchenne muscular dystrophy, spinal muscular atrophy, and severe hepatic veno-occlusive disease.

The high percentage of orphan drugs being approved by the FDA is welcome news for the 30 million Americans who have a rare disease, said Peter L. Saltonstall, president and CEO of NORD.

“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 7000 rare diseases still have no treatment options,” he said in a statement. “We look forward to still more progress in 2017 and beyond.”

While the overall number of drugs approved in 2016 was lower than the previous year, the majority (73%) of those that were approved benefitted from one of the FDA’s programs to expedite the process of drug review, such as breakthrough therapy designation or priority review designation. In addition, nearly all (95%) of new products were approved during the “first cycle” without any delays to approval, John Jenkins, MD, who recently retired from his position as director of the Office of New Drugs at the FDA, wrote in a blog post.

“Moreover, 86% of the novel drug approvals were approved in the U.S. before they were approved by any other regulatory authorities,” he wrote.

Even though there were fewer drugs approved than the average 29 approvals per year over the last decade, the way drugs were approved show that the Center for Drug Evaluation and Research is reviewing drugs as quickly as possible.

“We thank the FDA for its continued flexibility in reviewing applications that often include small studies due to the small number of patients with the disease,” Saltonstall said.