FDA surpassed previous record, approved 21 new orphan drugs in 2015

The Food and Drug Administration in 2015 approved 21 new orphan drugs for rare diseases, the organization announced recently. This group of drugs made up 47% of all approvals in 2015, making it the year that the most orphan drugs were approved in FDA history. 

 

For the National Organization for Rare Disorders, a nonprofit representing patients and families affected by rare diseases, the record number of approvals is evidence of progress in treatment options. 

 

“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,” NORD president and CEO Peter Saltonstall 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. … We look forward to still more approvals in 2016 and beyond.”