Gene Therapies: Safety, CMC Draft Guidances Coming From US FDA In 2018
Lengthy safety registries, extensive premarket testing for replication competent retrovirus, and manufacturing submission advice likely; pending CBER documents informed by review and approval of Novartis' Kymriah, Kite's Yescarta, and Spark's Luxturna.
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Accelerated approval would help agency monitor gene therapies for durability and off-target effects, US FDA commissioner says.
Scale-up in particular is a challenge and FDA wants to help lower the barrier, CBER Director Peter Marks tells rare disease conference.