약학회지

ISSN 0377-9556 (PRINT)
ISSN 2383-9457 (ONLINE)

Table. 1.

Table. 1. Comparative overview of AAV gene therapy approvals-FDA and EMA
Trade Name Generic Name AAV Serotype Transgene Sponsor Indication FDA Application Approval EMA Application Approval
Luxturna voretigene neparvovec AAV2 RPE65 Spark Therapeutics (US)
Novartis (EU)
RPE65mutation-associated retinal dystrophy 2017.07
2017.12
2017.07
2018.11
Zolgensma onasemnogene abeparvovec AAV9 SMA AveXis, Inc. (US)
Novartis (EU)
Spinal Muscular Atrophy with bi-allelic mutations on SMN1 gene 2018.10
2019.05
2018.10
2020.05
Hemgenix etranacogene dezaparvovec AAV5 Factor IX CSL Behring Hemophilia B (congenital Factor IX deficiency) 2022.03
2022.11.
2022.03
2023.02
Upstaza eladocagene exuparvovec AAV2 dopa decarboxylase (DDC) PTC Therapeutics aromatic L-amino acid decarboxylase (AADC) deficiency N/A 2020.01.
2022.07
Roctavian valoctocogene roxaparvovec AAV5 Factor VIII BioMarin Hemophilia A N/A 2021.06.
2022.08

Three AAV gene therapy drugs have received approvals from both the FDA and EMA, while an additional two drugs have been exclusively approved by EMA as of February 2023.

N/A: non-applicable

Yakhak Hoeji 2023;67:342-53 https://doi.org/10.17480/psk.2023.67.6.342
© 2023 Yakhak Hoeji