FDA approved more first-in-class drugs, gave more accelerated approvals in 2021

Regulatory NewsRegulatory News | 07 January 2022 |  By 

The US Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) approved 50 new drug therapies in 2021. While the figure is a slight decrease from 2020, more drugs were designated as first-in-class and used the accelerated approval pathway than in previous years.
The approvals reported by CDER do not include biologics license applications (BLAs) approved by FDA’s Center for Biologics Evaluation and Research (CBER). In 2021, CBER approved 10 different biological products, including the COVID-19 vaccine Comirnaty, for a total of 60 novel therapeutics approvals.
“Throughout 2021, the COVID-19 pandemic continued to present significant challenges to our entire staff. In spite of these hardships, we have approved many therapies that will advance health for the American public,” CDER director Patrizia Cavazzoni, MD, wrote in the center’s recent annual report.
Of the 50 drugs approved by CDER in 2021, 38 drugs (76%) were approved first in the United States, the FDA said in the report. First-in-class drugs comprised 27 of 50 drugs (54%) approved in 2021 compared with 21 of 53 first-in-class drugs (39.6%) in 2020, the agency noted. This represents an upward trend in first-in-class drug approvals, as FDA had previously reported 20 drug approvals (42%) in 2019, 19 approvals (32%) in 2018, and 15 approvals (33%) in 2017 were given first-in-class designation.
Regarding orphan drugs, the agency said 26 of 50 drugs (52%) in 2021 were approved as orphan drugs for rare diseases. In previous years, the proportion of orphan drugs approved has been inconsistent, with FDA identifying 31 of 53 drugs (58.4%) in 2020, 21 of 48 drugs (43.7%) in 2019, 34 of 59 (57.6%) in 2018, and 18 of 46 drugs (39.1%) in 2018 as having been approved by CDER as orphan drugs.
Products were also approved across a variety of regulatory pathways, FDA said. More drugs used an expedited review method for approval as compared to previous years. Overall, 37 drugs (74%) in 2021 were approved through at least one expedited pathway, an increase from 68% of novel drugs in 2020, 60% in 2019, and 73% in 2018.
Fast Track status was designated to 18 of 50 drugs (36%) in 2021, a slight increase from 17 of 53 drugs (32%) in 2020 and 17 of 48 drugs (35.4%) in 2019, but a decrease from 24 of 59 drugs (40.6%) in 2018. CDER designated Breakthrough Therapy status for 14 of 50 drugs (28%) in 2021, a decrease from 22 of 53 drugs (41.5%) in 2020 but an increase from 13 of 48 drugs (27%) in 2019 and 14 of 59 drugs (23.7%) in 2018 designated as Breakthrough Therapies.
A higher percentage of novel drugs continued to use the Accelerated Approval pathway in 2021, according to the report, with 14 of 50 drugs (28%) using the pathway. In contrast, 12 of 53 drugs (22.6%) in 2020, 9 of 48 drugs (18.7%) in 2019, and 4 of 59 drugs (6.7%) in 2018 received the Accelerated Approval designation.
One of the most talked about drug approvals of the year was Aduhelm, a first-in-class treatment for patients with Alzheimer’s disease that was approved under the accelerated approval pathway. FDA’s Peripheral and Central Nervous System Advisory Committee nearly unanimously voted to recommend against approving Aduhelm in November 2020, and three committee members stepped down from the FDA advisory committee after the treatment was approved in June 2021. Critics of Aduhelm cited a lack of evidence on whether the treatment’s approval based on reduction of surrogate endpoint amyloid beta in the brain suggested a clinical benefit of the drug. In December 2021, the European Medicines Agency recommended against market authorization of Aduhelm, explaining “the link between this effect and clinical improvement had not been established.” (RELATED: FDA defends Aduhelm's accelerated approval, while others call for reform, Regulatory Focus 14 July 2021)
Other drugs approved by CDER in 2021 include treatments for HIV-1, type 2 diabetes, chronic kidney disease, inflammatory bowel disease, ulcerative colitis, Crohn’s disease, systemic lupus erythematosus, and various types of cancer, among a variety of treatment options for rare diseases.
New Drug Therapy Approvals 2021


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