top of page

NEWS & INSIGHTS

Dive into the Biopharma Space

Stay up-to-date on major biopharma news, deeper dives into specific companies and biopharma themes, and how it all relates to biopharma partners.

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


 

💡 Do you sell your products or services to biopharma companies? 💡


BiopharmIQ MarketCompass is a market intelligence tool to help with your business development. Get a free sample report of potential targets.



Article History:

Originally posted 8/2/23 (RB)


Comentarios


bottom of page