GENE THERAPY AUTHORIZATIONS AND ORPHAN DRUG DESIGNATIONS BY THE FDA AND EMA
Author(s)
Rosa Rodriguez-monguio, PhD, MS1, Anna Pelc, MS, MBA, MPH2, Mana Rahimian, PharmD, MS2, Enrique Seoane-Vazquez, PhD2.
1University of California San Francisco, San Francisco, CA, USA, 2Chapman University School of Pharmacy, Irvine, CA, USA.
1University of California San Francisco, San Francisco, CA, USA, 2Chapman University School of Pharmacy, Irvine, CA, USA.
OBJECTIVES: Gene therapies represent a paradigm change in the treatment and prevention of unmet medical needs, especially for patients with rare diseases and conditions. The objective of this study was to compare gene therapy authorizations, orphan designations and approvals, regulatory development and authorization pathways, and authorization time gaps between the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA).
METHODS: We conducted a descriptive comparative analysis using publicly available FDA and EMA regulatory data sources through June 1, 2026. Authorization time gaps were calculated as the difference between FDA and European Union marketing authorization dates.
RESULTS: Thirty-two gene therapies were authorized by at least one agency. The FDA authorized 29 therapies (90.6%), and the EMA authorized 24 (75.0%). The FDA was the first agency to authorize 22 therapies (68.8%), and the EMA was first for 10 (31.3%). The median authorization time gap between FDA and EMA authorizations was 310 days for gene therapies authorized by both agencies. Five EMA-authorized therapies (20.8%) had post-authorization market withdrawal, marketing discontinuation, or authorization expiration in the EU, and two FDA-authorized therapies (6.9%) were discontinued from the market in the U.S. Orphan designation had been granted to all 29 FDA-authorized gene therapies. However, 28 of 29 therapies (96.6%) were authorized for an orphan indication; nadofaragene was the only FDA-authorized gene therapies not approved for an orphan indication. Among 24 EMA-authorized gene therapies, 23 (95.8%) had orphan designation, but only 15 (62.5%) retained orphan status at authorization. The FDA granted 688 orphan designations for gene therapies, and the EMA granted 367 from 2000 to June 1, 2026.
CONCLUSIONS: Gene therapy authorizations differed across the FDA and EMA in timing, regulatory pathways, and market status. Most gene therapies were indicated for rare diseases, with many additional gene therapies for rare diseases currently under development.
METHODS: We conducted a descriptive comparative analysis using publicly available FDA and EMA regulatory data sources through June 1, 2026. Authorization time gaps were calculated as the difference between FDA and European Union marketing authorization dates.
RESULTS: Thirty-two gene therapies were authorized by at least one agency. The FDA authorized 29 therapies (90.6%), and the EMA authorized 24 (75.0%). The FDA was the first agency to authorize 22 therapies (68.8%), and the EMA was first for 10 (31.3%). The median authorization time gap between FDA and EMA authorizations was 310 days for gene therapies authorized by both agencies. Five EMA-authorized therapies (20.8%) had post-authorization market withdrawal, marketing discontinuation, or authorization expiration in the EU, and two FDA-authorized therapies (6.9%) were discontinued from the market in the U.S. Orphan designation had been granted to all 29 FDA-authorized gene therapies. However, 28 of 29 therapies (96.6%) were authorized for an orphan indication; nadofaragene was the only FDA-authorized gene therapies not approved for an orphan indication. Among 24 EMA-authorized gene therapies, 23 (95.8%) had orphan designation, but only 15 (62.5%) retained orphan status at authorization. The FDA granted 688 orphan designations for gene therapies, and the EMA granted 367 from 2000 to June 1, 2026.
CONCLUSIONS: Gene therapy authorizations differed across the FDA and EMA in timing, regulatory pathways, and market status. Most gene therapies were indicated for rare diseases, with many additional gene therapies for rare diseases currently under development.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
HPR6
Topic
Health Policy & Regulatory
Topic Subcategory
Approval & Labeling
Disease
Genetic, Regenerative & Curative Therapies