US Food and Drug Administration Authorizations of Therapeutic Biologics and Biosimilars, 1980-2024
Author(s)
Roya Hosseini, PharmD, PhD1, Rosa Rodriguez-monguio, PhD, MS2, Enrique Seoane-Vazquez, PhD3.
1Chapman University School of Pharmacy, Irvine, CA, USA, 2School of Pharmacy, University of California San Francisco, San Francisco, CA, USA, 3Professor, Chapman University School of Pharmacy, Irvine, CA, USA.
1Chapman University School of Pharmacy, Irvine, CA, USA, 2School of Pharmacy, University of California San Francisco, San Francisco, CA, USA, 3Professor, Chapman University School of Pharmacy, Irvine, CA, USA.
OBJECTIVES: Objectives: The FDA Center for Drug Evaluation and Research regulates new drugs and therapeutic biologics. This study assessed trends in the approval of therapeutic biologics license applications (BLA) and biosimilars approved by the US Food and Drug Administration (FDA) from 1980 to 2024.
METHODS: Methods: Regulatory information was collected from the FDA's Purple Book and Drugs@FDA databases. Trends in authorizations for new BLAs and biosimilars were analyzed using descriptive statistics.
RESULTS: Results: Between 1980 and 2024, the FDA approved 267 new BLAs, of which 241 (90.3%) were marketed in the US as of December 31, 2024. The average annual number ± standard deviation of new BLAs increased from 1.3±1.6 in the 1980s to 15.2±1.8 in the period 2020-2024. New BLAs represented 18.5% of total CDER approvals during the study period, increasing from 4.4% of CDER new drug approvals in the 1980s to 31.0% in 2020-2024. The therapeutic classes with the highest number of BLAs included Antineoplastic and Immunomodulating Agents (134, 50.2%), Alimentary Tract and Metabolism (32, 12.0%), and Blood and Blood-Forming Organs (22, 8.2%). Of the BLAs, 53.6% received orphan designation, 61.4% priority review designation, 20.9% accelerated approval, 35.5% fast-track designation, and 40.4% breakthrough therapy designation. 14 BLAs faced biosimilar competition authorized by the FDA. The FDA authorized 65 biosimilars, including 11 interchangeable biosimilars. The average ± standard deviation time from the first reference BLA to the first biosimilar authorization was 17.4 ± 13.8 years (median 19.1 years, interquartile range 5.3 years).
CONCLUSIONS: Conclusions: The number of BLAs licensed by the FDA increased significantly from 1980 to 2024. Most BLAs had orphan designation, were authorized using priority review, and benefited from regulatory pathways and designations designed to expedite development and FDA review. Only a small fraction of BLAs approved during the study period faced biosimilar competition.
METHODS: Methods: Regulatory information was collected from the FDA's Purple Book and Drugs@FDA databases. Trends in authorizations for new BLAs and biosimilars were analyzed using descriptive statistics.
RESULTS: Results: Between 1980 and 2024, the FDA approved 267 new BLAs, of which 241 (90.3%) were marketed in the US as of December 31, 2024. The average annual number ± standard deviation of new BLAs increased from 1.3±1.6 in the 1980s to 15.2±1.8 in the period 2020-2024. New BLAs represented 18.5% of total CDER approvals during the study period, increasing from 4.4% of CDER new drug approvals in the 1980s to 31.0% in 2020-2024. The therapeutic classes with the highest number of BLAs included Antineoplastic and Immunomodulating Agents (134, 50.2%), Alimentary Tract and Metabolism (32, 12.0%), and Blood and Blood-Forming Organs (22, 8.2%). Of the BLAs, 53.6% received orphan designation, 61.4% priority review designation, 20.9% accelerated approval, 35.5% fast-track designation, and 40.4% breakthrough therapy designation. 14 BLAs faced biosimilar competition authorized by the FDA. The FDA authorized 65 biosimilars, including 11 interchangeable biosimilars. The average ± standard deviation time from the first reference BLA to the first biosimilar authorization was 17.4 ± 13.8 years (median 19.1 years, interquartile range 5.3 years).
CONCLUSIONS: Conclusions: The number of BLAs licensed by the FDA increased significantly from 1980 to 2024. Most BLAs had orphan designation, were authorized using priority review, and benefited from regulatory pathways and designations designed to expedite development and FDA review. Only a small fraction of BLAs approved during the study period faced biosimilar competition.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
HPR61
Topic
Health Policy & Regulatory
Topic Subcategory
Approval & Labeling
Disease
No Additional Disease & Conditions/Specialized Treatment Areas