COMPARATIVE ANALYSIS OF GENE THERAPIES AUTHORIZED BY THE U.S. FOOD AND DRUG ADMINISTRATION AND THE EUROPEAN MEDICINES AGENCY
Author(s)
Mana Rahimian, PharmD, MS1, Anna Pelc, MS, MBA, MPH1, Lawrence M. Brown, PharmD, PhD; FAPhA1, Marc L. Fleming, BSPharm, MPH, MS, PhD1, Vaishali Shukla, MSc, MSRA, MS, PhD1, Rosa Rodriguez-monguio, PhD, MS2, Enrique Seoane-Vazquez, PhD1;
1Department of Pharmaceutical Economic and Policy, Chapman University School of Pharmacy, Irvine, CA, USA, 2Department of Clinical Pharmacy, School of Pharmacy, University of California San Francisco, San Francisco, CA, USA
1Department of Pharmaceutical Economic and Policy, Chapman University School of Pharmacy, Irvine, CA, USA, 2Department of Clinical Pharmacy, School of Pharmacy, University of California San Francisco, San Francisco, CA, USA
OBJECTIVES: To compare the regulatory characteristics of gene therapies authorized by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA).
METHODS: Gene therapy authorization data were extracted from the FDA and EMA websites as of December 31, 2025, and analyzed descriptively.
RESULTS: Thirty gene therapies were authorized: 19 (63.3%) by both agencies, 8 (26.7%) by the FDA only, and 3 (10.0%) by the EMA only. Antineoplastic and immunomodulating agents accounted for the most authorizations (12, 40.0%), followed by therapies for blood and blood-forming organs (6, 20.0%) and alimentary tract and metabolism disorders (3, 10.0%). The FDA authorized 27 gene therapy products, of which 21 (77.8%) received priority review, 5 (18.5%) fast track designation, 9 (33.3%) breakthrough therapy designation, 9 (33.3%) regenerative medicine advanced therapy designation, 14 (51.9%) accelerated approval, 24 (88.9%) orphan drug designation, and 5 (18.5%) rolling review. Fidanacogene elaparvovec was discontinued from the market. The EMA authorized 22 gene therapies, with one additional therapy receiving a positive Committee for Medicinal Products for Human Use (CHMP) opinion pending European Commission authorization. Five (22.7%) therapies were discontinued from the market. Among the 19 therapies authorized by both agencies, all indications differed in at least one element, including differences in disease characteristics (17, 89.5%), line-of-therapy (1, 5.3%), and age group (6, 31.6%). The FDA authorized most products first (14, 73.7%), with a median EMA authorization gap of 309.0 days (IQR, 281.0).
CONCLUSIONS: The majority of gene therapies were authorized by both agencies, with the FDA approving products earlier than the EMA by a median of less than one year. Most therapies targeted rare diseases and received expedite development and approval procedures. There were differences in the indications of gene therapies authorized by FDA and EMA.
METHODS: Gene therapy authorization data were extracted from the FDA and EMA websites as of December 31, 2025, and analyzed descriptively.
RESULTS: Thirty gene therapies were authorized: 19 (63.3%) by both agencies, 8 (26.7%) by the FDA only, and 3 (10.0%) by the EMA only. Antineoplastic and immunomodulating agents accounted for the most authorizations (12, 40.0%), followed by therapies for blood and blood-forming organs (6, 20.0%) and alimentary tract and metabolism disorders (3, 10.0%). The FDA authorized 27 gene therapy products, of which 21 (77.8%) received priority review, 5 (18.5%) fast track designation, 9 (33.3%) breakthrough therapy designation, 9 (33.3%) regenerative medicine advanced therapy designation, 14 (51.9%) accelerated approval, 24 (88.9%) orphan drug designation, and 5 (18.5%) rolling review. Fidanacogene elaparvovec was discontinued from the market. The EMA authorized 22 gene therapies, with one additional therapy receiving a positive Committee for Medicinal Products for Human Use (CHMP) opinion pending European Commission authorization. Five (22.7%) therapies were discontinued from the market. Among the 19 therapies authorized by both agencies, all indications differed in at least one element, including differences in disease characteristics (17, 89.5%), line-of-therapy (1, 5.3%), and age group (6, 31.6%). The FDA authorized most products first (14, 73.7%), with a median EMA authorization gap of 309.0 days (IQR, 281.0).
CONCLUSIONS: The majority of gene therapies were authorized by both agencies, with the FDA approving products earlier than the EMA by a median of less than one year. Most therapies targeted rare diseases and received expedite development and approval procedures. There were differences in the indications of gene therapies authorized by FDA and EMA.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
HPR117
Topic
Health Policy & Regulatory
Topic Subcategory
Approval & Labeling
Disease
SDC: Rare & Orphan Diseases, STA: Genetic, Regenerative & Curative Therapies, STA: Personalized & Precision Medicine