Characteristics of Cell and Gene Therapies Authorized By the Food and Drug Administration and the European Medicines Agency
Speaker(s)
Kamerikar V1, Dheer P1, Bermudez BL1, Shukla V1, Rodriguez-Monguio R2, Fleming M1, Seoane-Vazquez E1
1Chapman University School of Pharmacy, Irvine, CA, USA, 2University of California San Francisco, San Francisco, CA, USA
Presentation Documents
OBJECTIVES: Cell and gene therapies represent a promising shift in the treatment and prevention paradigm for cancer, genetic diseases, and other conditions lacking effective treatments. This study examined the characteristics of the market authorizations of cell and gene therapies by the European Medicines Agency (EMA) and the Food and Drug Administration (FDA).
METHODS: Data from the FDA and EMA websites as of December 31, 2023, was analyzed descriptively.
RESULTS: The FDA and EMA approved a total of 21 gene and 14 cell therapies. The FDA authorized 26 therapies and the EMA 24. The first two therapies were authorized in 2009, 15 therapies (1.5±0.7 per year) from 2010 to 2019, and 19 (4.8±1.9) from 2020 to 2023. Fifteen products were approved by both agencies, with 12 approved first by the FDA and a median EMA approval gap of 145 days (interquartile range 280 days). The EMA withdrew authorization for 3 gene and 4 cell therapies. The therapeutic classes with the largest number of authorizations were antineoplastic and immunomodulating agents (4, 40.0%), blood and blood-forming organs (6, 17.1%), alimentary tract and metabolism (4, 11.4%), and musculoskeletal system (4, 11.4%). Orphan designation was granted to 22 (84.6%) therapies by the FDA and 15(62.5%) by the EMA. The FDA used priority review for 21(80.8%) therapies, fast track for 12 (46.2%), breakthrough therapy for 15(57.7%), and regenerative medicine advanced therapy designation for 7(26.9%). The EMA used additional monitoring for 19 (79.2%) therapies, conditional approval for 9 (37.5%), exceptional circumstances for 3 (12.5%), and accelerated assessment for 1 (4.2%).
CONCLUSIONS: A significant number of new cell and gene therapies have been approved by both the FDA and EMA, mainly targeting rare cancers and genetic diseases. While the FDA often approved therapies first, majority of these therapies were approved by only one agency. Most therapies were authorized using expedited regulatory procedures.
Code
HTA64
Topic
Health Technology Assessment
Topic Subcategory
Systems & Structure
Disease
Biologics & Biosimilars, Drugs, Genetic, Regenerative & Curative Therapies