6 Pharmaceutical Technology REGULATORY SOURCEBOOK MARCH 2020 P h a r mTe c h . c o m
Gene and Cell Therapies
S
ince 2017, FDA has approved four gene therapies, and
regulators expect the number of gene and cell therapies
under development to increase dramatically. FDA has
already received more than 900 investigational new drug
(IND) applications for clinical studies involving these therapies (1).
FDA has published guidance on cell and gene therapies since 1998,
when the field was brand new, according to Kevin Hacker, director
of life sciences at Clarkston Consulting (2). To help spur innovation,
FDA offers cell and gene therapy developers a number of expedited
review options, including "breakthrough therapy" and "fast track."
In 2018, the agency introduced another category, "regenerative medi-
cine advanced therapy," and published six draft guidance documents
on the following clinical and manufacturing topics:
• Gene therapies for hemophilia and retinal disorders, key focus
areas for many developers, as well as for rare diseases in general
• Testing of retroviral vector-based gene therapy products dur-
ing their manufacture and patient follow-up
• Long-term patient follow-up and monitoring for potential ad-
verse events after administration of gene therapy
• Chemistry, manufacturing, and control (CMC) information
that should be included in investigational new drug applica-
tions (IND) for gene therapies.
FDA updated and finalized these guidance documents on Janu-
ary 28, 2020, with the stated goal of supporting "safe innovation"
and offering developers "meaningful guidance" on complex clinical
questions (3). ipopba
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Stock.adobe.com
Inside FDA's New Gene and
Cell Therapy Guidances
Agnes Shanley
In January 2020, the agency
finalized six clinical
development and
manufacturing guidance
documents and drafted new
guidance on what would
qualify new gene therapies
as orphan drugs.
