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GRAFT-VERSUS-HOST DISEASE –
OPPORTUNITY ANALYSIS AND FORECASTS TO 2018
GRAFT-VERSUS-HOST DISEASE –
OPPORTUNITY ANALYSIS AND FORECASTS TO 2018
Executive Summary
Table above presents the key metrics for graft-
Key Metrics for Graft-Versus-Host Disease (GVHD)
in the Six Major Pharmaceutical Markets, 2013–
2018
versus-host disease (GVHD) in the six major
pharmaceutical markets (US, France, Germany,
2013 Epidemiology
First allogeneic HSCTs (men and women) (N)
6MM
15,991
Diagnosed incident cases of aGVHD in first
allogeneic HSCTs (men and women) (N), 6MM
8,062
Diagnosed incident cases of cGVHD in first
allogeneic HSCTs (men and women) (N), 6MM
7,359
Italy, Spain, and UK) covered in this report during
the forecast period from 2013–2018.
Steady Growth Expected in the US and EU
2013 Market Sales
GVHD Markets from 2013–2018
The GVHD market was valued at $297.0m across
US
$209.7m
5EU
$86.0m
the 6MM in 2013, and is expected to increase to
$295.7m
$409.0m in 2018, at a Compound Annual Growth
Total
Pipeline Assessment
Rate (CAGR) of 6.61%. Growth is anticipated to be
Number of drugs in Phase I–II
33
Number of first-in-class drugs in Phase I–IIb
26
Key Events (2013–2018)
Level of
Impact
2014 Food and Drug Administration (FDA)
approval and restricted* launch of Prochymal
(remestemcel-L) for aGVHD
↑↑
2015 European Medicines Agency (EMA)
approval and restricted* launch of Prochymal
for aGVHD
↑↑
2016 ATG-Fresenius (EZ-2053) launch
↑↑
2016 Budenofalk (budesonide) EU launch
slowest in the US, as there are fewer market
changes expected during the five-year forecast
period. In contrast, due to the multiple product
launches in Europe, growth in the 5EU is expected
to accelerate towards the end of the forecast
period, posting a CAGR of 9.42% during this
timeframe. In addition, multiple biologic products
↑
2018 Launch of Leukotac (inolimomab)
↑↑↑
2018 Launch of Begedina (anti-CD26)
↑↑↑
will fall off their respective patent cliffs during the
2013–2018 forecast period. However, this will not
impact the present GVHD forecast for two very
important reasons: First, biosimilar versions of key
2018 Market Sales
US
$273.0m
5EU
$134.0m
Total
$407.0m
Source: GlobalData
off-label
GVHD
(rituximab)
biologics,
such
as
Rituxan
and
Campath/Lemtrada
(alemtuzumab), are in a very early developmental
For the purposes of this report, the six major pharmaceutical markets =
US and 5EU (France, Germany, Italy, Spain, and UK).
stage and to date have failed clinical trials.
aGVHD = acute GVHD; cGVHD = chronic GVHD; HSTC =
hematopoietic stem cell transplantation
Secondly, hematologists across the 6MM are not
expected to use biosimilar versions of branded
biologics in GVHD patients whose health is already
severely compromised. Furthermore, key opinion
leaders
(KOLs)
interviewed
by
GlobalData
unanimously agreed that they would prefer for new
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GRAFT-VERSUS-HOST DISEASE –
OPPORTUNITY ANALYSIS AND FORECASTS TO 2018
Executive Summary
biosimilars to be tested in a GVHD clinical trial

setting before they use them in the clinic.
data on off-label second-line therapies in both
aGVHD and cGVHD, which will lead to
In total, six products are expected to enter the
increased
market by 2018. Osiris Therapeutics/Mesoblast’s
Bristol-Myers
2015 (5EU), reaching $12.2m in sales from the
known as ATG-F) is expected to enter the US
already-established
Squibb’s
(BMS’)
Orencia

Increasing numbers of HSCT patients.

A trend towards prescribing of biologics and
patented small molecules, which will increase
GVHD market, and these two products are
the GVHD market value.
estimated to generate 2018 sales of $11.9m and
$4.5m, respectively. Both Jazz Pharmaceuticals’
of
(abatacept).
6MM in 2018. Dr. Falk Pharma’s Budenofalk is
2017, while in the same year, ATG-Fresenius (also
uptake
treatments, such as Roche’s Rituxan and
Prochymal is expected to launch in 2014 (US) and
forecast to enter the European GVHD market in
Academic publication of more favorable clinical

A high incidence of cGVHD throughout the
Leukotac and Adienne Pharma’s Begedina are
2013–2018 forecast period, which will lead to
forecast to enter the GVHD market in 2018, with
an
Leukotac being launched only in the 5EU. Due to
prescriptions.
their anticipated premium pricing, Begedina and

increase
in
expensive
long-term
The release of company-funded randomized
Leukotac are expected to add over $18m to the
clinical trial data, which can be leveraged by
6MM sales in 2018.
reimbursement agencies to fund expensive
Major drivers of growth in the GVHD market over
biologics and patented small molecules.
the forecast period include:


T-regulatory (T-reg) cell and selective T-cell (T-
The launch of new therapies with orphan
lymphocyte) therapies currently developed in
designations. Since orphan drugs are entitled
academic institutes, which could be licensed
to premium pricing, the aGVHD market will
by pharmaceutical companies and drive their
accrue increased revenue.
clinical development.
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GRAFT-VERSUS-HOST DISEASE –
OPPORTUNITY ANALYSIS AND FORECASTS TO 2018
Executive Summary
Major barriers to the growth of the GVHD market

during the forecast period include:

in most EU countries could hinder future drug
uptake.
Reimbursement pressures in the EU could
lead to the funding of generic small molecules
The conservative approach to GVHD treatment

The complex pathophysiology of the disease
or low-efficacy drugs because the latter are
and the lack of adequate GVHD animal models
already in stock. For example, Johnson &
create
Johnson’s (J&J’s) Remicade (infliximab) is
development.
favored in the UK at the expense of Amgen’s
Enbrel (adalimumab), purely because the
former drug is what can be funded by the
National Health Service (NHS) at the moment,
significant
challenges during
drug
Figure below illustrates the global GVHD sales for
the six major markets (6MM) (US and 5EU) for all
three GVHD indications during the five-year
forecast period from 2013–2018.
as it is readily available.

The lack of national treatment guidelines in all
6MM could lead to a significant proportion of
patients being enrolled in clinical trials, thus
dissolving potential future GVHD revenues.
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GRAFT-VERSUS-HOST DISEASE –
OPPORTUNITY ANALYSIS AND FORECASTS TO 2018
Executive Summary
Global Sales for GVHD by Region and GVHD Indication, 2013–2018
2013 - Total GVHD sales $295.7m
3%
5%
5%
26%
10%
39%
6%
71%
35%
2018 - Total GVHD sales $407.0m
5%
3%
5%
36%
30%
11%
9%
67%
34%
US
France
Germany
Italy
Spain
UK
aGVHD
cGVHD
Prophylaxis
Source: GlobalData
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5
GRAFT-VERSUS-HOST DISEASE –
OPPORTUNITY ANALYSIS AND FORECASTS TO 2018
Executive Summary
Research and Development Strategies will
pipeline
Drastically Shape the GVHD Pipeline Over the
prophylaxis.
Next Five Years
In addition to the above strategies, blocking
Over the past two decades, the GVHD market has
communication between host antigen-presenting
been evolving, with the introduction of biological
cells and donor T cells has been a focus of R&D in
and patented small-molecule immunosuppressive
GVHD for over two decades. However, most of
off-label therapies being added to the treatment
these R&D ventures involve academically-run
paradigm. The current research and development
clinical studies. Targeting niche subgroups within
(R&D) programs in GVHD are exciting, with novel
GVHD, such as steroid-refractory aGVHD and
therapies (such as Adienne Pharma’s Begedina)
cGVHD, also emerges as one of the key R&D
and potential first-in-class treatments (such as
strategies in the GVHD pipeline, with 31 pipeline
mesenchymal
products targeting these populations.
stem
cell
[MSC]
therapies).
targeting
the
indication
of
GVHD
However, as a consequence of low funds accrual
Unmet Needs Remain Largely Unattained in
(as seen in clinical trial NCT00623012), low patient
GVHD Management
recruitment
(as
seen
in
clinical
trial
NCT00616954), and clinical trial failures (as seen
in clinical trial NCT00720850), the GVHD pipeline
has faced a period of slow development. Currently,
R&D strategies in GVHD are characterized by a
trend towards improving prophylactic regimens,
inhibiting T-cell proliferation and activation, using
treatments adopted from hematologic indications,
and targeting niche subgroups within the GVHD
population.
GVHD is a market with a substantial number of
unattained unmet needs. This is compounded by
the fact that there are no guidelines in place for the
management of GVHD; instead, there are only
recommendations for several therapies. This is
largely due to the orphan nature of the disease, but
it is mainly because of the lack of randomized
clinical trials and the use of variable clinical trial
endpoints in the studies that have been conducted
to date. This has resulted in clinical data that are
The current GVHD prophylactic regimens are
not comparable and/or not directly applicable to all
tailored to the type of conditioning and transplant
groups of GVHD patients, which, in turn, means
type used for HSCT. However, these therapies
that guidelines cannot be devised.
carry a risk of opportunistic infection and provide
inadequate GVHD prophylaxis. As a result, the
R&D focus in GVHD remains partly fixated on
prophylaxis. There are three products in the
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GRAFT-VERSUS-HOST DISEASE –
OPPORTUNITY ANALYSIS AND FORECASTS TO 2018
Executive Summary
Another crucial unmet need is the lack of GVHD-
In all, despite the ongoing development of products
specific therapies that induce durable remission.
and/or initiatives for the resolution of each unmet
Although the treatments that are currently used in
need, the GVHD market is a difficult one to
the management of GVHD (both acute and
introduce rapid changes into, as any changes
chronic) are established and have a good
could affect the survival outcomes for patients. As
reputation regarding their efficacy profiles for their
such, the highest priority is for more randomized
respective indication, they do not seem to offer
clinical trials to be conducted.
durable remission in the GVHD setting. One
example is the extracorporeal photophoresis (ECP)
The GVHD Market Provides an Abundance of
Opportunities
system, which does not have toxicities and can
induce a quick response, but this response is longlasting in only 20–30% of patients. Also, therapies
with minimal side effects are desired, since GVHD
patients are generally not physically fit and cannot
endure
additional
physical
stress
due
to
conditioning or prophylaxis drugs, or other drugs,
such as antibiotics, antivirals, and antifungal
therapies, that are used to treat or prevent the
There is a large opportunity for pharmaceutical
companies to conduct randomized clinical trials on
immunosuppressive therapies that are being used
off-label in the GVHD setting. This opportunity
could be exploited by supporting existing academic
organizations in their efforts to clarify the treatment
algorithm for GVHD in order to offer optimized
treatment outcomes for patients receiving HSCT.
Further incorporation of specific primary endpoints
other conditions that they develop.
in the clinical trial setting of aGVHD and cGVHD
Focusing on patient segments, the management of
steroid-refractory aGVHD (SR-aGVHD) patients
and GVHD patients with respiratory involvement is
challenging and becomes increasingly difficult with
increasing disease duration.
can
be
circumvented
prophylactic measures,
which
with
could
better
possibly
minimize the manifestation of GVHD. However, the
complex pathophysiology of GVHD, combined with
the challenge of personalized treatment design in
GVHD, renders a perfect prophylaxis unattainable
for the foreseeable future.
pharmaceutical industry. Implementing a more
robust set of endpoints will allow drug developers
to generate accurate and comparable data that
physicians can rely upon in order to adjust their
To some extent, most of the aforementioned unmet
needs
remains an opportunity for both academia and the
GVHD management protocols.
Although targeted and stratified clinical trials would
likely lead to drugs being approved for narrower
indications within the GVHD market, the upside is
that the medical community could come closer to a
much
needed
therapy
for
sclerotic
cGVHD
patients. Overall, there is a great need for
therapies with better safety and efficacy profiles
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GRAFT-VERSUS-HOST DISEASE –
OPPORTUNITY ANALYSIS AND FORECASTS TO 2018
Executive Summary
that are specific to GVHD pathophysiology and
Figure below provides GlobalData’s competitive
account for the nature of the disease. Still, there is
assessment of the GVHD prophylaxis landscape
room in the market for products for sclerotic
from 2013–2018.
cGVHD patients, particularly given the acute and
poor prognosis elements of GVHD.
Competitive Assessment of GVHD Prophylaxis OffLabel Therapies and Late-Stage Pipeline Agents,
2013–2018
Rituxan Remains the Protagonist of the GVHD
4.5
Market
Standard of care:
CsA+MTX
ATG-Fresenius
based prophylaxis
Prograf and
Rapamune instead
of CsA
and as such, the historic use of calcineurin
inhibitors with a short course of methotrexate
(MTX)
will
remain
prophylactic
regimen
the
most
through
avidly-used
2018.
Astellas
Pharma’s Prograf (tacrolimus) and anti-thymocyte
globulins (ATGs) are expected to also remain key
products for prophylaxis. They have been widely
COMMERCIAL SCORE
4.0
The prophylaxis setting of GVHD is conservative,
Rapamune+CsA
without MTX
Rituxan based
prophylaxis
Velcade based
prophylaxis
3.5
Lemtrada based
prophylaxis
Thymoglobuline
based prophylaxis
3.0
Orencia based
prophylaxis
2.5
MultiStem based
prophylaxis
2.0
2.0
2.5
3.0
3.5
CLINICAL SCORE
4.0
4.5
5.0
Source: GlobalData
CsA = cyclosporine A
MTX = methotrexate
used to date and are trusted by hematologists. In
GlobalData’s
the
SR-aGVHD prevails as the most urgent and poorly
prophylaxis landscape, Roche’s Rituxan emerged
managed population in the aGVHD setting, and as
as the most clinically and commercially attractive
such, there are plenty of innovative initiatives
prophylaxis regimen (see figure below). It is a
targeting this group. Because of the complexity of
favored therapy that is used in both aGVHD and
aGVHD
cGVHD prophylaxis due to its growing reputation in
immunotherapies and immunosuppressive small
the
Sanofi/Genzyme’s
molecules have and are being used for its
Campath/Lemtrada will remain an essential part of
management, giving way to a complex landscape
conditioning regimens, but not prophylaxis, due to
where establishing reputation among hematology
its highly infectious and generally harmful side
communities through trial funding and proven
effects. Over the 2013–2018 forecast period,
efficacy is key for an aGVHD therapy to move
increasing adverse reactions to classic therapy
forward.
field.
competitive
assessment
Meanwhile,
of
pathophysiology,
many
different
might give rise to Orencia-based prophylaxis, as
well as MultiStem (modified mesenchymal stem
cells)-based prophylaxis, which promises effective
prophylaxis with only one use.
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GRAFT-VERSUS-HOST DISEASE –
OPPORTUNITY ANALYSIS AND FORECASTS TO 2018
Executive Summary
Figure below provides GlobalData’s competitive
assessment of the aGVHD landscape from 2013–
Competitive Assessment of cGVHD Off-Label
Therapies and Late-Stage Pipeline Agents, 2013–
2018
2018.
5.0
Nipent Gleevec
Rituxan
4.5
5.0
Nipent
Leukotac
COMMERCIAL SCORE
mTOR inhibitors
Rituxan
4.5
COMMERCIAL SCORE
Competitive Assessment of aGVHD Off-Label
Therapies and Late-Stage Pipeline Agents, 2013–
2018
MMF
ECP
4.0 CD25α inhibitor
therapies
TNFα inhibitor
therapies
3.0
mTOR inhibitor
CD25α inhibitor
therapies
Lemtrada
2.5
2.5
3.0
3.5
CLINICAL SCORE
4.0
4.5
5.0
ECP = extracorporeal photophoresis; MMF = mycophenolate mofetil;
mTOR = mammalian target of rapamycin
Prochymal
Begedina
3.5
Source: GlobalData
Lemtrada
2.5
Standard of care:
Steroids
2.0
Thymoglobuline
3.0
ECP
4.0
2.0
Standard of care:
CsA + Steroids
3.5
MMF
2.0
2.0
2.5
3.0
3.5
4.0
CLINICAL SCORE
4.5
5.0
Source: GlobalData
X = Clinical Score; Y = Commercial Score
CsA = cyclosporine A; ECP = extracorporeal photophoresis; mTOR =
mammalian target of rapamycin; TNFα = tumor necrosis factor alpha
What Do Physicians Think?
In regard to which part of GVHD management
requires
The cGVHD market remains stagnant from the
improvement,
KOLs
interviewed
by
GlobalData offered the following:
innovation point of view. Initial therapy with
“I think the way to move forward [in the
steroids is the mainstay of treatment for cGVHD,
management of GVHD], clearly, is prophylaxis.
but because of the nature of steroid therapy —
That’s the way it is going to get better [in terms of
specifically, the severe side effects associated with
improved outcomes in HSCT].”
its long-term use — it cannot be used chronically.
[US] KOL, March 2014
Therefore, other treatments such as ECP, Rituxan,
Nipent
(pentostatin),
and
Gleevec
(imatinib
mesylate), are expected to face unhindered future
growth in the cGVHD setting during the 2013 to
2018 forecast period, and will emerge as the most
clinically and commercially attractive therapies in
GlobalData’s
competitive
assessment
of
cGVHD landscape, as shown in figure below.
the
“...actually,
the
major
point
in
this
[GVHD
management] is that when you start tapering
immunosuppressant therapy when you are already
using low doses of prednisone and [a] calcineurin
inhibitor, there is a time point where a subset of
these patients flare again with chronic GVHD.
There is no standard tapering protocol.”
[EU] KOL, March 2014
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GRAFT-VERSUS-HOST DISEASE –
OPPORTUNITY ANALYSIS AND FORECASTS TO 2018
Executive Summary
Commenting on the management of patients with
Discussing the efficacy of the late-stage pipeline
toxicities, a KOL said:
therapy, Therakos’ Uvadex (methoxsalen), an ECP
“I would say that for MSC therapies, ECP, and
therapy, a KOL said:
rituximab, I feel quite comfortable regarding [the]
“It’s a very safe treatment if we compare [it] to all
management of their toxicity, and then the rest of
older therapies, because with ECP you have an
drugs, actually, are hard to manage, and you have
immunosuppressive effect, but it’s a progressive
a quite high incidence of infections, which is not
and chronic immunosuppressive effect, not a
easy when you have a patient with pre-existing or
drastic one….Probably, it’s important to introduce
serious toxicities. You can’t help these patients;
very early this kind of treatment, and to not to
they usually succumb to organ failure.”
prolong [it] if there is no effect. So, it’s important to
[EU] KOL, March 2014
do either eight or 10 photophoresis cycles to know
if there is a response or not, and after, to switch to
Discussing
therapy,
the
safety
Osiris
of
late-stage
pipeline
Therapeutics/Mesoblast’s
another therapy. But probably, photophoresis has
an important place in the therapeutic setting in
Prochymal, a KOL said:
acute and in chronic [GVHD]. In chronic [GVHD],
“I mean, there are [a] number of potential safety
it’s definitely effective in cutaneous and ocular
issues about what their [MSC therapies’] impact is.
involvement, as well as in mucositis.”
I expect Prochymal will receive a black box
[EU] KOL, March 2014
warning regarding viral infection or tumor relapse
risk or issues with chimers. The honest truth is, if
you are applying them in a setting of prophylaxis,
Commenting on the efficacy of Roche’s Rituxan, a
KOL stated:
that is the most comfortable way — and there are
“I have a good experience with rituximab; that is
data out there to support mesenchymal stem cell
very effective for cutaneous involvement and in
therapy in prophylaxis — then the downsides of
sclerosis,
having GVHD probably outweigh any potential
expression[s] of chronic [GVHD] disease. It [also]
perceived risk of the product. If the product
has good results in ocular lesions and mucositis.”
which
are
worked, I wouldn’t be concerned about the
the
most
frequent
[EU] KOL, March 2014
potential risks of it, because having a product that
worked in that setting would outweigh any potential
risk, but I don’t think the product worked.”
[EU] KOL, March 2014
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GRAFT-VERSUS-HOST DISEASE –
OPPORTUNITY ANALYSIS AND FORECASTS TO 2018
Executive Summary
Discussing the challenges associated with the
available treatment options in the GVHD market, a
KOL said:
“The fact that there is such [a] big array of
immunosuppressive drugs [used off-label to treat
GVHD], it makes it difficult to decide [what
treatment to select, especially as] none of them
have been studied properly [within the GVHD
setting].”
[US] KOL, April 2014
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GRAFT-VERSUS-HOST DISEASE –
OPPORTUNITY ANALYSIS AND FORECASTS TO 2018
Table of Contents
1
1
2
3
Table of Contents
Table of Contents ..................................................................................................................... 12
1.1
List of Tables .................................................................................................................... 19
1.2
List of Figures ................................................................................................................... 23
Introduction ............................................................................................................................... 24
2.1
Catalyst............................................................................................................................. 24
2.2
Related Reports ................................................................................................................ 25
2.3
Upcoming Related Reports ............................................................................................... 25
Disease Overview ..................................................................................................................... 26
3.1
3.1.1
Etiology ......................................................................................................................... 26
3.1.2
Pathophysiology ............................................................................................................ 28
3.1.3
Classification and Prognosis ......................................................................................... 29
3.2
4
Etiology and Pathophysiology ........................................................................................... 26
Symptoms ......................................................................................................................... 31
Epidemiology ............................................................................................................................ 33
4.1
Disease Background ......................................................................................................... 33
4.2
Risk Factors and Comorbidities ........................................................................................ 34
4.3
Global Trends ................................................................................................................... 36
4.4
Forecast Methodology....................................................................................................... 37
4.4.1
Sources Used................................................................................................................ 39
4.4.2
Sources Not Used ......................................................................................................... 45
4.4.3
Diagnosed Incident Cases of HSCTs ............................................................................ 46
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GRAFT-VERSUS-HOST DISEASE –
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Table of Contents
4.4.4
Diagnosed Incident Cases of Autologous HSCTs .......................................................... 48
4.4.5
Diagnosed Incident Cases of Allogeneic HSCTs ........................................................... 49
4.4.6
Diagnosed Incident Cases of aGVHD ........................................................................... 50
4.4.7
Four-Year Diagnosed Prevalent Cases of aGVHD ........................................................ 50
4.4.8
Diagnosed Incident Cases of cGVHD............................................................................ 51
4.4.9
Five-Year Diagnosed Prevalent Cases of cGVHD ......................................................... 51
4.5
4.5.1
Diagnosed Incident Cases of HSCTs ............................................................................ 52
4.5.2
Diagnosed Incident Cases of Autologous HSCTs .......................................................... 54
4.5.3
Diagnosed Incident Cases of Allogeneic HSCTs ........................................................... 55
4.5.4
Diagnosed Incident Cases of aGVHD ........................................................................... 57
4.5.5
Four-Year Diagnosed Prevalent Cases of aGVHD ........................................................ 59
4.5.6
Diagnosed Incident Cases of cGVHD ............................................................................ 61
4.5.7
Five-Year Diagnosed Prevalent Cases of cGVHD ......................................................... 62
4.6
5
Epidemiological Forecast for GVHD (2013–2023) ............................................................. 52
Discussion ........................................................................................................................ 64
4.6.1
Epidemiological Forecast Insight ................................................................................... 64
4.6.2
Limitations of the Analysis ............................................................................................. 65
4.6.3
Strengths of the Analysis ............................................................................................... 66
Current Treatment Options ....................................................................................................... 67
5.1
Overview ........................................................................................................................... 67
5.2
Product Profiles – Major Therapies ................................................................................... 69
5.2.1
Methylprednisolone (numerous brand and generic names) ........................................... 69
5.2.2
Cyclosporine (numerous brands and generic names).................................................... 73
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GRAFT-VERSUS-HOST DISEASE –
OPPORTUNITY ANALYSIS AND FORECASTS TO 2018
Table of Contents
5.2.3
5.3
5.3.1
Anti-Thymocyte Globulin (ATG) Therapies .................................................................... 78
Product Profiles – Off-Label Therapies.............................................................................. 83
Biologics (Tumor Necrosis Factor (TNF)α Inhibitors, Interleukin-2 Receptor α (CD25)
Inhibitors, Co-Stimulatory Blockers and Other Biologics) .............................................. 83
5.3.2
Small Immunosuppressive Molecules (mTOR Inhibitors, Solid Organ Transplant
Therapies, and Anti-Neoplastic Therapies) ................................................................. 102
6
Unmet Need and Opportunity ................................................................................................. 117
6.1
Overview ......................................................................................................................... 117
6.2
Lack of Randomized Clinical Trials ................................................................................. 120
6.2.1
Unmet Need ................................................................................................................ 120
6.2.2
Gap Analysis ............................................................................................................... 120
6.2.3
Opportunity ................................................................................................................. 121
6.3
No Consensus Regarding Clinical Trial Endpoints .......................................................... 121
6.3.1
Unmet Need ................................................................................................................ 121
6.3.2
Gap Analysis ............................................................................................................... 121
6.3.3
Opportunity ................................................................................................................. 122
6.4
Optimization of Prophylactic Measures ........................................................................... 122
6.4.1
Unmet Need ................................................................................................................ 122
6.4.2
Gap Analysis ............................................................................................................... 123
6.4.3
Opportunity ................................................................................................................. 123
6.5
Lack of Standardized Protocols for Established and Off-Label Therapies ....................... 124
6.5.1
Unmet Need ................................................................................................................ 124
6.5.2
Gap Analysis ............................................................................................................... 124
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Table of Contents
6.5.3
6.6
Opportunity ................................................................................................................. 125
Improved Treatment Outcomes for Patients Who Develop Toxicities .............................. 125
6.6.1
Unmet Need ................................................................................................................ 125
6.6.2
Gap Analysis ............................................................................................................... 126
6.6.3
Opportunity ................................................................................................................. 126
6.7
Desire for Treatments that Exhibit Longer-Lasting Efficacy Profiles ................................ 126
6.7.1
Unmet Need ................................................................................................................ 126
6.7.2
Gap Analysis ............................................................................................................... 127
6.7.3
Opportunity ................................................................................................................. 128
6.8
Management of SR-aGVHD Patients .............................................................................. 128
6.8.1
Unmet Need ................................................................................................................ 128
6.8.2
Gap Analysis ............................................................................................................... 129
6.8.3
Opportunity ................................................................................................................. 129
6.9
Management of Sclerotic cGVHD Patients ...................................................................... 129
6.9.1
Unmet Need ................................................................................................................ 129
6.9.2
Gap Analysis ............................................................................................................... 130
6.9.3
Opportunity ................................................................................................................. 130
6.10
Improved Prognosis for Patients with Lung Involvement ................................................. 131
6.10.1 Unmet Need ................................................................................................................ 131
6.10.2 Gap Analysis ............................................................................................................... 131
6.10.3 Opportunity ................................................................................................................. 132
7
Research and Development Strategies ................................................................................... 133
7.1
Overview ......................................................................................................................... 133
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Table of Contents
7.1.1
Improvement of Prophylactic Regimens ...................................................................... 133
7.1.2
Inhibition of T-Cell Proliferation and Activation ............................................................ 134
7.1.3
Adopting Treatments from Hematologic Indications..................................................... 135
7.1.4
Targeting Niche Subgroups Within GVHD ................................................................... 136
7.2
7.2.1
Current Clinical Trial Design............................................................................................ 137
Current Trial Designs are Dependent on the Stage Within the Treatment Algorithm That
a Product is Targeting ................................................................................................ 137
7.2.2
Lack of Consensus on Clinical Endpoints in Current Trial Designs .............................. 138
7.2.3
Patient Exclusion Issues in Current Trial Designs ....................................................... 138
7.2.4
Current Trial Design of Key Pipeline Products ............................................................. 138
7.3
Future Clinical Trial Design ............................................................................................. 140
7.3.1
Future Trial Designs Need to Incorporate Randomization ........................................... 140
7.3.2
Future Trial Designs Need to Incorporate Conditioning Regimens and Hematological
Patients ...................................................................................................................... 141
7.3.3
Companies Behind Off-Label Therapies Should Strategize to Conduct Randomized
Studies ....................................................................................................................... 141
7.3.4
Design of Early-Phase Clinical Trials for Cellular and Gene Therapy Products
Accommodates 2014 Guidance from the FDA ............................................................ 141
8
Pipeline Assessment............................................................................................................... 142
8.1
Overview ......................................................................................................................... 142
8.2
Promising Drugs in Clinical Development........................................................................ 143
8.2.1
Leukotac (inolimomab) ................................................................................................ 143
8.2.2
Begedina (BT 5/9) ....................................................................................................... 148
8.2.3
Budenofalk (budesonide) ............................................................................................ 151
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Table of Contents
8.2.4
ATG-Fresenius (EZ-2053) ........................................................................................... 154
8.2.5
Prochymal (remestemcel-L) ........................................................................................ 157
8.2.6
MultiStem (modified mesenchymal stem cells) ............................................................ 162
8.2.7
Uvadex (Extracorporeal Photophoresis) ...................................................................... 165
8.2.8
orBec (beclomethasone dipropionate) ......................................................................... 169
8.3
9
Innovative Early-Stage Approaches ................................................................................ 172
8.3.1
Targeting Regulatory T Cells ....................................................................................... 175
8.3.2
Mesenchymal Stem Cell Therapies ............................................................................. 176
8.3.3
Immunomodulatory Cell Surface Receptor Inhibitors ................................................... 177
8.3.4
IL-6 Inhibitors .............................................................................................................. 178
Pipeline and Off-Label Valuation Analysis............................................................................... 179
9.1
Clinical Benchmarking of Key Pipeline and Off-Label Drugs ........................................... 179
9.1.1
GVHD Prophylaxis ...................................................................................................... 179
9.1.2
Acute GVHD................................................................................................................ 182
9.1.3
Chronic GVHD ............................................................................................................ 186
9.2
Commercial Benchmarking of Key Pipeline and Off-Label Drugs .................................... 187
9.2.1
GVHD Prophylaxis ...................................................................................................... 188
9.2.2
Acute GVHD................................................................................................................ 190
9.2.3
Chronic GVHD ............................................................................................................ 192
9.3
Competitive Assessment ................................................................................................. 194
9.3.1
GVHD Prophylaxis ...................................................................................................... 194
9.3.2
Acute GVHD................................................................................................................ 195
9.3.3
Chronic GVHD ............................................................................................................ 197
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GRAFT-VERSUS-HOST DISEASE –
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Table of Contents
9.4
Top-Line Five-Year Forecast........................................................................................... 199
9.4.1
US ............................................................................................................................... 204
9.4.2
5EU ............................................................................................................................. 208
10 Appendix................................................................................................................................. 212
10.1
Bibliography .................................................................................................................... 212
10.2
Abbreviations .................................................................................................................. 231
10.3
Methodology ................................................................................................................... 237
10.4
Forecasting Methodology ................................................................................................ 237
10.4.1 Diagnosed GVHD Patients .......................................................................................... 237
10.4.2 Percent Drug-Treated Patients .................................................................................... 238
10.4.3 Drugs Included in Each Therapeutic Class .................................................................. 238
10.4.4 Launch and Patent Expiry Dates ................................................................................. 239
10.4.5 General Pricing Assumptions ...................................................................................... 239
10.4.6 Individual Drug Assumptions ....................................................................................... 240
10.4.7 Generic Erosion .......................................................................................................... 247
10.4.8 Pricing of Pipeline Agents............................................................................................ 247
10.5
Physicians and Specialists Included in This Study .......................................................... 248
10.6
About the Authors ........................................................................................................... 250
10.6.1 Author ......................................................................................................................... 250
10.6.2 Reviewer ..................................................................................................................... 250
10.6.3 Epidemiologist ............................................................................................................. 251
10.6.4 Global Head of Healthcare .......................................................................................... 251
10.7
About GlobalData............................................................................................................ 252
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Table of Contents
10.8
1.1
Disclaimer ....................................................................................................................... 252
List of Tables
Table 1: Steps Involved in the Development of aGVHD.................................................................................. 28
Table 2: Steps Involved in the Development of cGVHD .................................................................................. 29
Table 3: Grading System of Organ Involvement in aGVHD ............................................................................ 30
Table 4: Classification of Organ Involvement in cGVHD ................................................................................. 31
Table 5: Symptoms of aGVHD ....................................................................................................................... 31
Table 6: Symptoms of cGVHD ....................................................................................................................... 32
Table 7: Risk Factors and Comorbidities for GVHD ........................................................................................ 35
Table 8: 6MM, Sources of HSCT, aGVHD, and cGVHD Data Used for the Forecast ...................................... 39
Table 9: 6MM, Sources Not Used in the Epidemiological Analysis of GVHD ................................................... 46
Table 10: 6MM, Diagnosed Incident Cases of HSCTs, Both Sexes, All Ages, N, 2013–2023 .......................... 53
Table 11: 6MM, Diagnosed Incident Cases of Autologous HSCTs, Both Sexes, All Ages, N, 2013–2023 ....... 54
Table 12: 6MM, Diagnosed Incident Cases of Allogeneic HSCTs, Both Sexes, All Ages, N, 2013–2023 ......... 56
Table 13: 6MM, Diagnosed Incident Cases of aGVHD in Diagnosed Incident Cases of First Allogeneic HSCTs,
Both Sexes, All Ages, N, 2013–2023 .............................................................................................. 58
Table 14: 6MM, Four-Year Diagnosed Prevalent Cases of aGVHD in Diagnosed Incident Cases of First
Allogeneic HSCTs, All Ages, Both Sexes, N, 2013–2023................................................................ 60
Table 15: 6MM, Diagnosed Incident Cases of cGVHD in Diagnosed Incident Cases of First Allogeneic HSCTs,
All Ages, Both Sexes, N, 2013–2023 .............................................................................................. 61
Table 16: 6MM, Five-Year Diagnosed Prevalent Cases of cGVHD in Diagnosed Incident Cases of First
Allogeneic HSCTs, All Ages, Both Sexes, N, 2013–2023................................................................ 63
Table 17: Product Profile – Methylprednisolone ............................................................................................. 70
Table 18: Efficacy of Methylprednisolone in GVHD Studies ............................................................................ 71
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Table of Contents
Table 19: SWOT analysis – Methylprednisolone, 2014................................................................................... 73
Table 20: Product Profile – Cyclosporine ....................................................................................................... 75
Table 21: Efficacy of Cyclosporine in GVHD Studies ...................................................................................... 76
Table 22: SWOT Analysis – Cyclosporine, 2014 ............................................................................................ 77
Table 23: Product Profiles – ATG Therapies .................................................................................................. 80
Table 24: Efficacy of ATG Therapies in GVHD ............................................................................................... 81
Table 25: SWOT Analysis – ATG Therapies, 2014 ......................................................................................... 82
Table 26: Product Profile – Biologics – TNFα Inhibitors .................................................................................. 85
Table 27: Efficacy of Anti-TNFα Therapies in GVHD ...................................................................................... 86
Table 28: Product Profiles – Biologics – IL-2Rα (CD25) Inhibitors .................................................................. 89
Table 29: Efficacy of IL-2Rα (CD25) Inhibitor Therapies in GVHD .................................................................. 90
Table 30: Product Profile – Biologics – Co-Stimulatory Blockers .................................................................... 93
Table 31: Efficacy of Co-Stimulatory Blocker Therapies in GVHD................................................................... 94
Table 32: Product Profile – Other Biologics (Campath/Lemtrada and Rituxan) ............................................... 96
Table 33: Efficacy of Other Biologic Therapies (Campath/Lemtrada and Rituxan) in GVHD ............................ 97
Table 34: Safety Profile – Off-Label Biologics Used in GVHD, 2014 ............................................................... 99
Table 35: SWOT Analysis – Biologics – TNFα Inhibitors, 2014 ..................................................................... 100
Table 36: SWOT Analysis – Biologics – IL-2Rα (CD25) inhibitors, 2014 ....................................................... 101
Table 37: SWOT Analysis – Biologics – Co-Stimulatory Blockers, 2014 ....................................................... 101
Table 38: SWOT Analysis – Biologics – Others, 2014 .................................................................................. 102
Table 39: Product Profile – Small Immunosuppressive Molecules – mTOR Inhibitors ................................... 104
Table 40: Efficacy of mTOR Inhibitors in GVHD ........................................................................................... 105
Table 41: Product Profile – Small Immunosuppressive Molecules – SOT Therapies ..................................... 106
Table 42: Efficacy of SOT Therapies in GVHD ............................................................................................. 107
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Table of Contents
Table 43: Product Profile – Small Immunosuppressive Molecules – Anti-Neoplastic Therapies..................... 110
Table 44: Efficacy of Anti-Neoplastic Therapies in GVHD ............................................................................. 111
Table 45: Safety Profile – Small Immunosuppressive Molecules .................................................................. 113
Table 46: SWOT Analysis – Small Immunosuppressive Molecules – mTOR Inhibitors, 2014 ........................ 114
Table 47: SWOT Analysis – Small Immunosuppressive Molecules – SOT Therapies, 2014.......................... 115
Table 48: SWOT analysis – Small Immunosuppressive Molecules – Anti-Neoplastic Therapies, 2014 .......... 116
Table 49: Unmet Need and Opportunity in GVHD ........................................................................................ 119
Table 50: Clinical Trial Design of Key Pipeline Drugs for GVHD, July 2014 .................................................. 139
Table 51: GVHD – Late Stage Pipeline, 2013 .............................................................................................. 143
Table 52: Product Profile – Leukotac (inolimomab) ...................................................................................... 145
Table 53: Efficacy of Leukotac in GVHD ...................................................................................................... 147
Table 54: SWOT Analysis – Leukotac (inolimomab), 2014 ........................................................................... 148
Table 55: Product Profile – Begedina (BT 5/9) ............................................................................................. 149
Table 56: Efficacy of Begedina in GVHD ...................................................................................................... 150
Table 57: SWOT Analysis – Begedina (BT 5/9), 2014 .................................................................................. 151
Table 58: Product Profile – Budenofalk (budesonide) ................................................................................... 152
Table 59: SWOT Analysis – Budenofalk, 2014 ............................................................................................. 153
Table 60: Product Profile – ATG-Fresenius (EZ-2053) ................................................................................. 155
Table 61: Efficacy of ATG-Fresenius in GVHD (EZ-2053) ............................................................................ 156
Table 62: SWOT Analysis – ATG-Fresenius, 2014 ....................................................................................... 157
Table 63: Product Profile – Prochymal (remestemcel-L) ............................................................................... 160
Table 64: SWOT Analysis – Prochymal (remestemcel-L), 2014.................................................................... 162
Table 65: Product Profile – MultiStem (modified mesenchymal stem cells) ................................................... 163
Table 66: SWOT Analysis – MultiStem (modified mesenchymal stem cells) ................................................. 164
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Table of Contents
Table 67: Product Profile – Uvadex (Extracorporeal Photophoresis) ............................................................. 167
Table 68: Efficacy of Uvadex in GVHD......................................................................................................... 168
Table 69: SWOT Analysis – Uvadex (Extracorporeal Photophoresis) ........................................................... 169
Table 70: Product Profile – orBec (beclomethasone dipropionate)................................................................ 171
Table 71: SWOT Analysis – orBec (beclomethasone proprionate), 2014 ...................................................... 172
Table 72: Early-Stage Pipeline Products for GVHD, July 2014 ..................................................................... 174
Table 73: Clinical Benchmarking for GVHD Prophylaxis, 2014 ..................................................................... 181
Table 74: Clinical Benchmarking for aGVHD, 2014 ...................................................................................... 185
Table 75: Clinical Benchmarking for cGVHD, 2014 ...................................................................................... 187
Table 76: Commercial Benchmarking for GVHD Prophylaxis, 2014 .............................................................. 189
Table 77: Commercial Benchmarking for aGVHD, 2014 ............................................................................... 191
Table 78: Commercial Benchmarking for cGVHD, 2014 ............................................................................... 193
Table 79: Top-Line Sales Forecasts ($m) for GVHD, 2013–2018 ................................................................. 201
Table 80: Key Events Impacting Sales for GVHD, 2013–2018...................................................................... 203
Table 81: Global GVHD Market – Drivers and Barriers, 2013–2018 ............................................................. 204
Table 82: Key Launch Dates, GVHD, 2013–2018 ........................................................................................ 239
Table 83: Key Patent Expiries, GVHD, 2013–2018....................................................................................... 239
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GRAFT-VERSUS-HOST DISEASE –
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Table of Contents
1.2
List of Figures
Figure 1: Schematic of HSCT ...................................................................................................................... 27
Figure 2: Schematic Flow Chart of the Derivation of the aGVHD and cGVHD Patient Population in the 6MM
.................................................................................................................................................... 37
Figure 3: 6MM, Diagnosed Incident Cases of HSCTs , Both Sexes, All Ages, N, 2013–2023 ....................... 53
Figure 4: 6MM, Diagnosed Incident Cases of Autologous HSCTs, Both Sexes, All Ages, N, 2013–2023 ...... 55
Figure 5: 6MM, Diagnosed Incident Cases of Allogeneic HSCTs, Both Sexes, All Ages, N, 2013–2023 ...... 57
Figure 6: 6MM, Diagnosed Incident Cases of aGVHD in Diagnosed Incident Cases of First Allogeneic
HSCTs, Both Sexes, All Ages, N, 2013–2023 ............................................................................... 59
Figure 7: 6MM, Four-Year Diagnosed Prevalent Cases of aGVHD in Diagnosed Incident Cases of First
Allogeneic HSCTs, All Ages, Both Sexes, 2013–2023................................................................... 60
Figure 8: 6MM, Diagnosed Incident Cases of cGVHD in Diagnosed Incident Cases of First Allogeneic
HSCTs, All Ages, Both Sexes, N, 2013–2023 ............................................................................... 62
Figure 9: 6MM, Five-Year Diagnosed Prevalent Cases of cGVHD in Diagnosed Incident Cases of First
Allogeneic HSCTs, All Ages, Both Sexes, N, 2013–2023 .............................................................. 63
Figure 10: Trends in aGVHD Management in the 6MM, 2014......................................................................... 68
Figure 11: Trends in cGVHD Management in the 6MM, 2014 ......................................................................... 69
Figure 12: Competitive Assessment of GVHD Prophylaxis Off-Label Therapies and Late-Stage Pipeline
Agents, 2013–2018 .................................................................................................................... 195
Figure 13: Competitive Assessment of aGVHD Off-Label Therapies and Late-Stage Pipeline Agents, 2013–
2018 ........................................................................................................................................... 197
Figure 14: Competitive Assessment of cGVHD Off-Label Therapies and Late-Stage Pipeline Agents, 2013–
2018 ........................................................................................................................................... 199
Figure 15: Global Sales for GVHD by Region and GVHD Indication, 2013–2018 .......................................... 202
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GRAFT-VERSUS-HOST DISEASE –
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Introduction
2
Introduction
Graft-versus-host disease (GVHD) is an orphan indication with a poor prognosis and high mortality
rates. It is a complication of hematopoietic stem cell transplantation (HSCT), and takes different
forms, depending on the time of disease manifestation and organ localization. As GVHD
pathogenesis remains largely elusive, the current treatments used for its management are not
GVHD-specific, but rather, are off-label therapies borrowed from immunological and hematological
indications. However, the GVHD pipeline is rich and diverse, and promises a big change in the
landscape of the GVHD market.
2.1
Catalyst
Despite the vast array of available off-label therapies (approximately 30) for the treatment and/or
prevention of GVHD, very few of these therapies have been tested in large randomized clinical
trials. This has resulted in vague treatment recommendations and many patients being enrolled in
institutional
clinical
trials.
The
only
available
standard
of
care
is
intravenous
(IV)
methylprednisolone. However, it fails to produce a complete response (CR) in more than 50% of
treated patients. As a result, steroid-refractory patient subgroups face a poor prognosis, with a
deteriorating quality of life (QoL). This problem is further compounded by the fact that second- and
third-line treatments can vary from country to country across the six major markets (6MM) (US,
France, Germany, Italy, Spain, and UK), and also between different medical institutions in the
same country.
Over GlobalData’s 2013–2018 forecast period, the main drivers of growth in the GVHD market
include the increasing numbers of allogeneic HSCTs and the increasing use of marketed and offlabel biologic therapies across the 6MM.
Sanofi/Genzyme is a key player in the GVHD market, with EU-approved Thymoglobulin (antithymocyte globulin [rabbit]) and the off-label biologic therapy, Campath/Lemtrada, which infiltrate
the prophylaxis, acute GVHD (aGVHD), and chronic GVHD (cGVHD) setting. Still, Johnson &
Johnson’s (J&J’s) Remicade (infliximab) has gained a significant share of the aGVHD patient
group, as it is one of the few drugs that is effective in GVHD with gastrointestinal (GI) involvement.
Meanwhile, gaining more and more ground in the cGVHD setting is Roche’s Rituxan (rituximab),
which GlobalData estimates will be one of the best-selling biologics by value in GVHD, in the US by
2018.
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GRAFT-VERSUS-HOST DISEASE –
OPPORTUNITY ANALYSIS AND FORECASTS TO 2018
Introduction
2.2

Related Reports
GlobalData (2014). EpiCast Report: Graft-Versus-Host Disease – Epidemiology Forecast to
2023, June 2014, GDHCER064-14

GlobalData (2014). OpportunityAnalyzer: Chronic Lymphocytic Leukemia – Opportunity
Analysis and Forecasts to 2018, June 2014, GDHC017POA

GlobalData (2014). PharmaPoint: Ulcerative Colitis – Global Drug Forecast and Market
Analysis to 2022, February 2014, GDHC80PIDR

GlobalData (2014). PharmaPoint: Crohn’s Disease – Global Drug Forecast and Market
Analysis to 2022, January 2014, GDHC77PIDR

GlobalData (2013). OpportunityAnalyzer: Acute Myeloid Leukemia (AML) – Opportunity
Analysis and Forecasts to 2017, August 2013, GDHC003POA

GlobalData (2013). PharmaPoint: Chronic Myeloid Leukemia (CML), Global Drug Forecast and
Market Analysis to 2022, April 2013, GDHC103PIDR
2.3

Upcoming Related Reports
GlobalData (2014). PharmaSphere – Emerging Biotechnologies: Stem Cell Therapy, August
2014, GDHC012PSR

GlobalData (2014). OpportunityAnalyzer: - Non Hodgkin B cell Lymphoma, Opportunity
Analysis and Forecasts to 2018, July 2014, GDHC035POA
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GRAFT-VERSUS-HOST DISEASE –
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Appendix
10.7
About GlobalData
GlobalData is a leading global provider of business intelligence in the healthcare industry.
GlobalData provides its clients with up-to-date information and analysis on the latest developments
in drug research, disease analysis, and clinical research and development. Our integrated business
intelligence solutions include a range of interactive online databases, analytical tools, reports, and
forecasts. Our analysis is supported by a 24/7 client support and analyst team.
GlobalData has offices in New York, San Francisco, Boston, London, India, Korea, Japan,
Singapore, and Australia.
10.8
Disclaimer
All Rights Reserved.
No part of this publication may be reproduced, stored in a retrieval system, or transmitted in any
form by any means, electronic, mechanical, photocopying, recording, or otherwise, without the prior
permission of the publisher, GlobalData.
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252