8 BioPharm International eBook March 2020 www.biopharminternational.com
Inside FDA's New Gene and Cell
Therapy Guidance
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.
S
ince 2017, FDA has approved four gene ther-
apies, 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 thera-
pies 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 ther-
apy developers a number of expedited review options,
including "breakthrough therapy" and "fast track." In
2018, the agency introduced another category, "regenera-
tive medicine advanced therapy," and published six draft
guidances on the following topics:
• Gene therapies for hemophilia and retinal disor-
ders, as well as for rare diseases in general
• Testing retroviral vector-based gene therapy prod-
ucts during manufacture and patient follow-up
• Long-term patient follow-up and monitoring for
adverse events after treatment with gene therapy
• Chemistry, manufacturing, and control (CMC)
information that should be included in investi-
gational new drug applications (IND) for gene
therapies.
FDA updated and finalized these guidance documents
on January 28, 2020, with the stated goal of support-
ipopba
-
Stock.adobe.com
AGNES SHANLEY
Regulatory Sourcebook Gene and Cell Therapies
