Spark's Early Christmas Present: US FDA Approves Luxturna For Vision Loss
Review and approval of first directly administered gene therapy targeting a disease caused by genetic mutations embodies 'spirit' of agency's forthcoming series of guidance documents on gene therapies, FDA Commissioner Gottlieb says; documents are likely to encourage development of the kind of novel and clinically meaningful endpoint that Spark used in its clinical program.
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Keeping Track: Novartis Scores Big Ahead Of US Memorial Day With Approvals For Gene Therapy Zolgensma, Oncologic Piqray
The latest drug development news and highlights from our US FDA Performance Tracker.
Trial protocols, annual reports, and adverse event reports on gene therapies will only go to US FDA under now-finalized amendments, a sign that the product category is maturing and can be regulated like other drugs and biologics.
CBER Director Peter Marks says the "half-life" for the existing six gene therapy guidances likely is two to three years due to the fast pace of scientific discovery.