LS&HC Horizons 2023 - Flipbook - Page 7
Hogan Lovells | 2023 Life Sciences and Health Care Horizons | Precision and Regenerative Medicine
Product sameness considerations for cell, tissue,
and gene therapy products
Whether a product is the “same” as a previously
approved product has broad implications on
a number of important regulatory decisions,
including exclusivity awards and approval
actions. Cell, tissue, and gene therapy (CTGT)
products challenge FDA’s existing regulatory
framework and its traditional notions of
product sameness.
In 2021, FDA issued a final guidance regarding
the sameness determination for gene therapy
products in the orphan drug context, which
established a general framework based on
both the transgene and vector. However, the
inherent complexity of gene therapy products
means that this general standard must be
tailored on a case-by-case basis.
New insight into FDA’s sameness analysis was
provided with the licensure of the chimeric
antigen receptor (CAR)-T product, Breyanzi®
(lisocabtagene maraleucel). In particular, FDA
determined that Breyanzi® was not the same as
a previously licensed CAR-T product for orphan
drug purposes even though both products use
Gary Veron
Partner, Washington, D.C.
Bryan Walsh
Associate, Washington, D.C.
a lentiviral vector encoding a CD19-specific
CAR with the same co-stimulatory domain.
FDA based its conclusion on the fact that
there are differences between the products
in the transgene hinge and transmembrane
sequences, which impact CAR-T cell function.
FDA also noted that the final cell compositions
of the products are different, and Breyanzi® is
administered at a defined ratio of T cell subsets.
This decision, particularly the analysis of
cellular function and T cell subset composition,
is an important benchmark for an increasingly
complex class of products.
We continuously monitor new developments
in this evolving area, and advise clients
regarding whether and how to engage with
FDA to achieve favorable policies, guidance,
and decisions.
Jason F. Conaty
Counsel, Washington, D.C.
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