USA – FDA: 2019 Continues Uptick in Orphan Drug Approvals

Similar to 2017 and 2018, the US Food and Drug Administration (FDA) in 2019 approved an increasing number of orphan drugs and biologics, as well as drugs targeted at specific cancers.

Sponsors have continued to target the development of pricier orphan treatments and the Center for Drug Evaluation and Research (CDER) reported approving more than twice as many orphan drugs in the past eight years as in the previous eight years (see graph below). In addition, more than one-third of CDER’s novel drug approvals in 2019 were cancer treatments.

“Patients with rare diseases frequently have few or no drugs available to treat their condition — and for them, approvals of so-called ‘orphan’ drugs can mean new hope for an enhanced quality of life, and in some cases, survival,” CDER Director Janet Woodcock said.