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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