Enter 2015 during which the FDA approved 45 new drugs, four more than in 2014 again the highest number since 1996.
FDA Drug Approvals (1994 through 2015). NME=New Molecular Entity, BLA=Biologic License Application;
Twenty-one (21) of those (47%) were “orphan” drugs. This compares to 17 orphan drugs approved in 2014 or 41% of the total.
Until a few years ago, if you asked a pharmaceutical company executive why his or her company developed and marketed an “orphan drug”—i.e., a drug for a disorder affecting fewer than 200,000 people in the U.S.—you would likely have gotten a response such as “because there is an unmet medical need” or something similar. Today, however, orphan drugs also have the potential to turn into blockbusters.
That potential is being realized. As reported recently by CNBC (http://cnb.cx/1kUViSf),
Orphan drug approvals are very profitable for the pharma industry, which often submits drugs to the FDA as orphan drugs but once approved, the drugs are “used broadly off-label with the lucretive orphan drug protections and exclusivity benefits,” according to the authors of a study from Johns Hopkins University School of Medicine recently published in the American Journal of Clinical Oncology (http://sco.lt/4pauhN).
Seven of the top 10 drugs in global sales have received an orphan designation from the FDA. Crestor, for example, received an orphan drug designation for the treatment of pediatric homozygous familial hypercholesterolemia in 2014 (see Table 1, below).