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AAV vector safety - FDA Insight (May ‘23)

Category: FDA & gene therapy safety

Opportunity: Proactively address FDA issues re: AAV vector safety

Trigger event: May 2023 FDA AdComm

Target Companies: Gene therapy sponsors and safety vendors

The FDA in its May 2023 Advisory Committee Briefing Document, provided helpful insights into its safety review of filings for AAV vector-based gene therapy products. The Briefing Document was for Sarepta’s SRP-9001 (delandistrogene moxeparvovec) gene therapy for Duchene muscular dystrophy (DMD) (FIG. 1). This FDA insight is helpful information for sponsors as well as vendors whose tools address these issues. The FDA cited 3 types of safety issues (FIG. 1):

1) Hepatoxicity

2) Thrombotic microangiopathies

3) Oncogenicity due to integration and insertional mutagenesis

See FIG. 2 and the citations therein for more details.

FIG. 1 FDA Briefing Doc Cover Page

FIG. 2 FDA’s list of Toxicities Associated with AAV Vector-Based Gene Therapy Products


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Article History:

Originally posted 8/2/23 (RB)


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