FDA: 2019 Continues Uptick in Orphan Drug Approvals

Regulatory NewsRegulatory News | 06 January 2020 |  By 

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.


As far as first-in-class therapies, CDER pointed to 20 of the 48 novel drugs approved in 2019 (42%), which is consistent with years past as CDER identified 19 approvals (32%) as first-in-class in 2018, 15 (33%) in 2017, eight (36%) in 2016 and 16 (36%) in 2015.

In addition, sponsors’ use one or more expedited programs, such as the fast track, breakthrough, priority review and/or accelerated approval, remained consistently high. 2019 saw 60% of CDER’s novel drug approvals (29 of 48) use one or more expedited programs, which compares with 43 of the 59 novel drug approvals in 2018 (73%) and 28 of 46 (60%) novel approvals in 2017.

First-cycle approvals have also lingered around 90% of all approvals for each of the past three years. By comparison, only 12% of generic drugs were approved in the first review cycle from FY2015 to 2017.

And 33 of the 48 novel drugs approved in 2019 (69%) were approved in the US ahead of other countries, which is slightly less than the 42 (71%) from 2018 and the 36 (78%) approved first in the US in 2017.

CDER also met all its PDUFA goal dates in each of the past three years. In late 2019, FDA also approved five new drugs months ahead of when they were expected to be approved.

New Drug Therapy Approvals 2019

FY 2019 Report from CBER’s Director


© 2023 Regulatory Affairs Professionals Society.

Discover more of what matters to you

No taxonomy