LS&HC Horizons 2022 - Flipbook - Page 9
Hogan Lovells | 2022 Life Sciences and Health Care Horizons
9
Precision and Regenerative Medicine
Product sameness in an increasingly complex environment: Considerations for cellular
and gene therapy products
Whether a product is the “same” as a previously
approved product impacts a number of important
regulatory decisions, including exclusivity awards and
approval actions. Increasingly complex and innovative
products in the cellular and gene therapy fields have
challenged FDA’s traditional sameness standards, but
the agency has made strides in addressing this gap.
• I n 2021, FDA finalized a guidance document
regarding the determination of sameness for gene
therapy products for rare diseases, for purposes of
orphan-drug designation and exclusivity. Although
the guidance provides that sameness is based on
transgene and vector characteristics, the inherent
complexity of gene therapy products means that this
general standard will continue to be refined on a
case-by-case basis.
• F
or biosimilar products, a showing of similarity to
the reference biological product is a critical first step
towards approval. Here, again, cellular and gene
therapy products raise novel issues for FDA and
sponsors to navigate. For example, CAR-T therapies
involve harvesting a patient’s T cells and genetically
modifying them ex vivo by retroviral transduction
to express a chimeric antigen receptor (CAR). The
CAR-T cells are then expanded and infused back into
the patient. FDA recently issued a draft guidance
document regarding the development of CAR-T
products, which discusses principles to show high
similarity after a manufacturing change. It remains to
be seen how FDA will implement those principles to
usher in the next generation of biosimilar products.
The evolving regulatory landscape requires that cellular
and gene therapy product manufacturers consider
whether and how to engage with regulators to assist in
the development of favorable policies and guidance.
David M. Fox
Partner, Washington, D.C.
George O’Brien
Partner, Washington, D.C.
Gary Veron
Partner, Washington, D.C.
