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manufacturing, distribution, and clinical trial analysis. In doing
so, the intent is to improve the time-to-market for life saving
therapeutics without compromising patient safety or drug efficacy.
Looking at biosimilars - has their growth/approval
rate met your expectations? If not, what has been
holding them back?
Treichler: Various biosimilars continue to advance through the
development pipeline in anticipation of innovators' products coming
off patent. The uptake in the market has been variable and depends
on the indication, and the policies that regulatory bodies have put
into place regarding interchangeability and substitutions. One key
challenge to a biosimilar gaining approval is proving its biosimilarity
to the reference product (the innovator product). Another challenge
for companies advancing biosimilar products is that upon launch, they
are likely to need to compete for market share not only with both the
innovator's product, but also with other biosimilars.
Vap: To date, 25 cell and gene therapies (CGT) have been approved
by the FDA. Looking at the growth/approval rate in 2022, bluebird
bio had two gene therapies approved - Zynteglo for treatment
of patients with beta-thalassemia and Skysona for treatment of
patients with cerebral adrenoleukodystrophy. Janssen also had a
cell therapy approved in 2022 - Carvykti for treatment of patients
with relapsed or refractory multiple myeloma. While three CGT
approvals in 2022 is a small fraction of the 25 total CGTs approved,
the industry is trending steadily and preparing to grow rapidly over
the coming years. In the 2022 Prescription Drug User Fee Act (PDUFA)
VII commitment letter, the FDA's Center for Biologics Evaluation
and Research (CBER) calls for additional resources to support the
increasing volume of CGT programs in the Office of Therapeutic
Products (OTP). In 2023 they plan to hire 132 additional resources
and another 48 in 2024. This level of resource recruitment comes
in light of bluebird bio's two 2022 approved gene therapies and in
preparation for an increasing amount in 2023, one of which may be
Vertex and CRISPR Therapeutics' ex vivo CRISPR/Cas9 gene-edited
therapy, exagamglogene autotemcel, for the treatment of sickle cell
disease and transfusion-dependent beta-thalassemia.
Supply chain issues have affected every industry.
Specifically, how has this issue impacted the
biopharmaceutical market?
Treichler: The supply chain issues caused by the pandemic and other
factors have led to inconsistency in the ability to source key raw
materials and consumables, for example purification resins. Long lead
times and stock outs can impact the ability to schedule manufacturing
slots, which can delay production. To mitigate this risk, many
companies have secured secondary supply sources, extended their
buying horizons, and increased their inventory of critical materials.
Some have also developed improved business analytics to identify and
track potential supply risks earlier, giving them more time to respond
to potential issues.
Vap: While supply chain issues have influenced drug development
and manufacturing timelines, there have also been other ways in
which the biopharmaceutical industry has been affected. Novel
techniques to reduce the need for more starting material have been
more widely adopted. Examples include plasmid amplification and
polymerase chain reaction (PCR) based mRNA manufacturing. The
latter of the two replaces a pDNA linearization approach to mRNA
manufacturing, requiring a significant amount of starting pDNA and
a master cell bank. To juxtapose the more novel methods to emerge
from supply chain constraints, there has also been a resurgence
of more traditional techniques to avoid the need for customized
solutions that supply chains are struggling to support. An example
of this is the use of CHO rather than HEK293 or SF9 platforms for the
production of gene therapies. CHO platforms have been proven as a
robust manufacturing method for monoclonal antibodies and many
single use system vendors have the necessary equipment, packaging,
and configurations for a CHO production system. While single use
vendors also support HEK293 and SF9 platforms, sometimes these
require custom solutions that strain the supply chain.
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