The CPhI Worldwide expert panel predicts pharma’s biggest
opportunities and threats in 2017 – the wider industry, attendees and
exhibitors increasingly look to the CPhI global events as a key indicator
of future trends and partnerships. The panel forecasts that with the
traditional blockbuster drug era widely considered to have passed,
orphan drugs and neglected diseases are, in the short-term, likely
to deliver pharma’s best revenue opportunities. Over the medium
term, developing world economies and cost reductions from new
technologies and working practices like Quality by Design (QbD) and
continuous processing should help sustain profits.
Alexei A. Yeliseev
Human cannabinoid receptor CB2
is an integral membrane protein
that belongs to a large superfamily of seven transmembrane domain G
protein-coupled receptors (GPCR). CB2
is a part of the endocannabinoid
system that regulates immune response, inflammation and pain
sensitivity. The rational development of novel specific pharmaceuticals
targeting this receptor relies on high resolution studies of the structure
and mechanisms of activation of CB2
. Here we review the methodology
for recombinant expression and purification of CB2
, suitable for
preparation of multi-milligram quantities of functionally active
receptor. CB2
is expressed in a functional form in E. coli, solubilized in
detergent micelles, and purified by tandem affinity chromatography.
The recombinant CB2
is amenable to functional and structural studies
by nuclear magnetic resonance spectroscopy and a wide range of
biochemical and biophysical techniques.
Igor Gorsky, Chris M. Hanf, Jeffrey Hartman, Dr. Mike Long, MBB
This is the third article in a series of publications titled “How Clean
is Clean in Drug Manufacturing?” The first and the second articles
introduced the term Equipment Cleaning Process (ECP); examined
residue limits setting, including health based considerations; and,
showed applicability to Cleaning Validation Practices of the Threestage
Process Validation Concepts described in the FDA Process
Validation Guidance. This article will continue the discussion about
a risk based and lifecycle approach to Cleaning Validation. We will
discuss assessment of variation in ECP and tools for benchmarking
the culture of Cleaning Validation at drug manufacturing facilities. In
a recent article, Dr. Mike Long asked a simple question: “How much
variation is acceptable in our products and processes?” Although the
question is simple, answering it could be very complex. It requires
understanding of product, process, and those factors that impact
variability. Very early on in the lifecycle of any process, including ECP,
risk tools should be used to help understand and control the amount
of variation.
Portable, handheld, and miniature spectrometers are ubiquitous
in the marketplace now. The possibility of instituting a raw material
identification and verification system in your supply chain management
system that meets regulatory and quality requirements is now possible
for every large, medium, and small pharmaceutical manufacturer and
compounding pharmacy. Cost considerations are no longer a barrier
as the costs for these devices are very affordable such that the return
on investment can be realized in a years’ time.
BASF creates chemistry for a sustainable future offering intelligent
solutions to the pharmaceutical industry. With our expertise in polymer
chemistry, R&D-capabilities and our commitment in developing
excipients, BASF continuously creates solutions for Instant & Modified
Release, Solubilization, Softgels, Skin-Delivery and Biologic Solutions.
BASF is also the leading supplier of selected APIs such as ibuprofen and
omega-3.
Saly Romero-Torres, Ph.D., James Moyne, Madhav Kidambi
It has been almost 15 years since the creation of the 21st Century
cGMP initiative by the Food and Drug Administration (FDA). The main
goal of this initiative was to encourage the adoption of innovative
manufacturing technologies within the pharmaceutical industry.1
Stefan R. Schmidt
In the last two years five novel fusion proteins were approved by the
FDA, raising the total number of marketed therapeutic fusion proteins
to thirteen. Several more are in late stage clinical trials and will probably
reach the market soon. This underlines a success story that started in
1998 with the approval of Enbrel® the first fusion protein ever. Fusion
proteins are generated by artificially joining two or more genes
that originally code for separate proteins by genetic engineering.
The result is a single polypeptide with functional properties of both
parental proteins. These combinations of unrelated domains do not
occur in nature and are solely based on intelligent human design.1
Often the components of fusion proteins are derived from different
cell types or cellular locations that do not necessarily fit together in
terms of physicochemical properties such as isoelectric point, charge
densities and hydrophobicity which ultimately lead to manufacturing
challenges. Here, the three areas, design, upstream and downstream
processing, which contribute mostly both to the challenges and the
respective solutions, are discussed (Figure 1).
Can you summarize briefly what the Catalent
Applied Drug Delivery Institute’s non-invasive
macromolecule delivery consortium (NMDC) has
achieved since it was established two years ago?
Vito Pirrera
Though not an afterthought by any means, validation is seldom
integrated into every phase of serialization project planning,
particularly the early stages, when the project’s scope and objectives
are being defined. Thinking about validation at the project’s inception
means the test scripts and documents that follow are more aligned
with the manufacturer’s requirements. It all starts with the important
Validation Master Plan. As its name implies, it lays the groundwork for
how the system is to be qualified.
Dr. Berthold Boedeker, Dr. Jørgen Magnus
Recent advances in developing new technologies such as the use of
disposables, closed systems operation and continuous processing
have an important impact on the production of biologics and their
manufacturing facilities resulting in much faster, less expensive and
simpler plant design, start-up and operation.1-3 This paper describes
the current status and limitations of using disposables and continuous
processing. In addition a case study of a flexible ballroom facility for
the production of monoclonal antibodies is shown, which is based on
in single use systems and continuous processing.
Emilie Branch
A patient-focused approach to the development of a drug is not a
new concept in the pharmaceutical industry, but it is an evolving
practice. Human factors engineering (HFE) - also known as usability
engineering, cognitive ergonomics, or user-centered design - is equal
parts psychology and engineering. HFE is also helping improve the
patient experience and smooth the path to personalized medications.
The actual definition of HFE continues to evolve and change from
year to year and industry to industry; however, the International
Ergonomics Associate (IES) adopted the following overarching
definition: “Ergonomics (or human factors) is the scientific discipline
concerned with the understanding of interactions among humans and
other elements of a system, and the profession that applies theory,
principles, data, and other methods to design in order to optimize
human well-being and overall system performance.”1
Harshada Sant, MS, Amitkumar Lad, PhD, Hemant N. Joshi, PhD, MBA
The purpose of this column is to highlight
and to summarize recent key patents in
the pharmaceutical arena issued by the US
Patent Office in November, 2016.