FDA sped progress for most of 2020's novel drugs

Regulatory NewsRegulatory News
| 14 January 2021 | By Kari Oakes 

Most novel drugs approved by the US Food and Drug Administration (FDA) in 2020 made their way through the approvals process using one or more expedited approval pathways, and over half of sponsors received the incentives that accompany orphan drug designation, according to an FDA report detailing the year’s approvals.
Novel drug approvals continued at a brisk clip, averaging over one per week in 2020. Despite the pandemic the 53 novel drugs approved in 2020 bested 2019’s 48 approvals but fell short of the 59 approved in 2018.
In the report on FDA’s novel drug approvals for 2020, FDA noted that 40%, or 21 of 53, approved drugs are considered first-in-class. The agency pulled out Rukobia (fostemsavir) as an example of a new antiretroviral medication in heavily pretreated individuals with HIV. Koselugo (selumetinib), approved to treat neurofibromatosis type 1, was also cited by the agency as a notable first-in-class therapy.
Rare diseases were addressed by 31 of the novel drugs, or 58%. Sponsors of these drugs received incentives under the Orphan Drug Act. In particular, FDA called out the first oral therapy for spinal muscular atrophy and an oral treatment for hereditary angioedema, as well as Lampit (nifurtimox) for Chagas disease. The latter received orphan designation despite the fact that the drug was developed in the 1960s.
Similarly, artesunate, developed in the 1970s, was approved for the first time in the US to treat severe malaria, which befalls about 300 US residents yearly.
In addition to the incentives offered through the orphan drug program, FDA also made use of several approval pathways that increase FDA-sponsor communication, boost flexibility for the sponsor, and shorten review timelines. In all, two thirds of the novel drugs approved in 2020 (36 of 53, or 68%) were approved by moving through at least one expedited development and review category.
About a third of 2020’s novel approvals (17 of 53, or 32%) were made through the Fast Track designation, which “speeds new drug development and review by increasing the level of communication between FDA and drug developers, and by enabling CDER to review portions of a drug application ahead of the submission of the complete application,” noted FDA.
All but five of the drugs that received Fast Track designation also had orphan designation.
Breakthrough therapy designation is reserved for drugs “for serious or life-threatening diseases for which there is unmet medical need and for which there is preliminary clinical evidence demonstrating that the drug may result in substantial improvement on a clinically significant endpoint,” wrote FDA. Nearly half (22 of 53, or 42%) of the novel drugs received breakthrough therapy status, which provides sponsors with all Fast Track benefits plus additional FDA guidance to ensure an efficient development program.
Priority review was granted to over half (30 of 53, or 57%) of novel drugs in 2020, meaning that the agency assessed them as potentially being able to “provide a significant advance in medical care.” FDA clarified that the novel drugs which fell into this category did not include those who received priority review by virtue of redemption of a Priority Review Voucher.
Under the accelerated approval program, about a quarter (12 of 53, or 23%) of the novel drugs were approved under a scheme that allows more flexibility in determining endpoints for clinical trials. The accelerated approval pathway is reserved for drugs offering a benefit over treatments that are currently available to address a condition that is serious or life-threatening.
Overall, 75% of the novel drugs, or 40 of the 53, were approved in the US before they received approval elsewhere in the world.


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