FDA Approving Orphan Drugs at a Record Pace

August 15, 2017Alerts FDA Alert

It is only August, but the U.S. Food and Drug Administration (FDA) is on track to set a record for orphan drugs approved this year. At last count, 42 orphan drugs have been approved between January 1, 2017 and August 9, 2017.

At its current pace, the FDA will approve more than 60 orphan drugs by the end of the year. That is 20 percent above its last record-breaking period in 2014 (49 approvals) and 2015 (48 approvals).

Figure 1. Orphan Drug Approvals by the FDA per Year


As you can see in the figure above, there has been a steady upwards trend in orphan drug approvals since the initiation of the Orphan Drug Act in 1983.

Each year, the number of drugs being given an orphan drug designation is also growing exponentially. In each of the past two years, more than 300 drugs were designated as orphan drugs by the FDA. And for the first seven months of 2017, 309 drugs have been designated as orphan drugs.

Figure 2. Orphan Drug Designations by the FDA per Year


An orphan drug designation gives companies numerous tax breaks and filing fee exemptions during the review process. In addition, if a drug gets approved, the company receives seven years of market exclusivity for that drug. With the addition of the pediatric rare disease priority review voucher, it is likely that even more companies will understand the financial and altruistic rewards that come from helping those in the rare disease community.

For more information about this alert, please contact Jean W. Frydman, who heads the firm's FDA Practice, at 609.895.6630 or [email protected].