LSHC Horizons Brochure 2024 - Flipbook - Page 24
Hogan Lovells | 2024 Life Sciences and Health Care Horizons | Cell, Tissue, and Gene Therapies
Medical device regulatory considerations for novel cell-based gene
therapies, Casgevy and Lyfgenia, FDA approved as biologics
On 8 December 2023, FDA approved two
groundbreaking cell-based gene therapies,
Casgevy and Lyfgenia, for treating sickle
cell disease (SCD) in patients 12 years and
older. While Casgevy utilizes CRISPR/Cas9
gene editing technology to modify patients’
hematopoietic stem cells (HSCs), Lyfgenia
uses a lentiviral vector to treat SCD in patients
with a history of vaso-occlusive events. Both
therapies are approved for autologous use only,
meaning healthy stem cells are collected from
the patient’s body, stored, and reinfused in a
single dose to the donating patient. Casgevy
and Lyfgenia are regulated as biological
products (biologics) by the Food and Drug
Administration (FDA); however, ancillary
products and components used in preparing
or processing the biologics are often regulated
as standalone medical devices or combination
products, depending on their intended use.
The process of collecting stem cells involves
mobilizing stem cells from a patient’s bone
marrow into their bloodstream, separating
them from other blood components using
an “apheresis machine”, and subsequently
modifying them by gene editing and
cryopreserving them to maintain viability
until transplantation. Blood collection
devices (e.g., tubes, centrifuges, and filter
systems) and blood administration sets used
to prepare autologous substances, with no
specific indications for use, have historically
been regulated by FDA’s Center for Devices
and Radiological Health (CDRH) through the
510(k) premarket pathway.
Randy J. Prebula
Partner
Washington, D.C.
In contrast, blood collection bags, automated
blood cell separators, and centrifuges labeled
specifically for use in processing substances,
have mostly been regulated by FDA’s Center
for Biologics Evaluation and Research (CBER)
as a standalone drug or biological product.
Moreover, if the gene therapies were packaged
or labeled for use with, for example, a catheter
used to attain vascular access for apheresis
(a device), FDA would likely regulate the
product as a biologic-led combination product.
Therefore, considering the type of ancillary
product/component and its manner of use,
a manufacturer may be required to comply
with additional FDA regulations beyond those
applicable to biologics.
Key Takeaways
FDA’s approval of Casgevy and Lyfgenia
provides important precedent and regulatory
clarity for emerging cell-based gene therapies
regulated as biological products. Nevertheless,
it is essential that manufacturers stay apprised
of potential regulatory hurdles involved in
developing future products. Understanding
the complexities of how ancillary products and
components are regulated will help ensure
manufacturers remain compliant with relevant
pre- and postmarket FDA regulations, while
also helping to ensure safe, seamless, and
effective delivery of these innovative therapies.
Sanchita R. Bose
Associate
Washington, D.C.
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