The U.S. Food and Drug Administration has announced 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.
Saltonstall added, "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."
"NORD has been the leading advocate for the rare disease community for more than three decades. The progress reflected in so many new therapies re-energizes all of us who are dedicated to improving the lives of people with rare diseases. We look forward to still more approvals in 2016 and beyond," Saltonstall concluded.
For more details about 2015's approvals, visit the Novel New Drugs Summary 2015.