GDHC103PIDR Chronic Myeloid Leukemia sample · PDF fileChronic Myeloid Leukemia (CML) Global Drug Forecast and Market Analysis to 2022 GDHC103PIDR / Published May 2013

Chronic Myeloid Leukemia (CML) Global Drug Forecast and Market Analysis to 2022

GDHC103PIDR / Published May 2013

Executive Summary

© GlobalData. This report is a licensed product and is not to be copied, reproduced, shared or resold in any form. Page 2 GDHC103PIDR / Published MAY 2013

CML: Key Metrics in Seven Major Pharmaceutical Markets 2012 Epidemiology

10-year Prevalent Population 78,697

Drug-Treated Population 74,575

2012 Market Sales

US $1,598m

5EU $926.8m

Japan $488.7m

Total $3,013m

Pipeline Assessment

Overall Strength of Pipeline Very Weak

Most Promising New Market Entrants Peak-Year Sales

Iclusig [ponatinib (Ariad)] $563.3m

Bosulif [bosutinib (Pfizer)] $133.9m

Key events (2012–2022) Level of Impact

Launch of Iclusig in the US/5EU in 2013, Japan in 2016 ↑↑

Launch of generic imatinib in the 7MM 2014–2016 ↓↓↓

Iclusig brand extension for newly diagnosed CP CML ↑

Launch of generic dasatinib in the 7MM 2020–2021 ↓↓↓

2022 Market Sales

US $1,026m

5EU $528.3m

Japan $564.7m

Total $2,119m Source: GlobalData 5EU = France, Germany, Italy, Spain, and UK; 7MM = US, 5EU, and Japan.

Sales for CML by Region Will Decline between 2012–2022

The CML therapeutics market in the 7MM was valued at

$3.013 billion in 2012. The US accounted for 53% of the

global market due to its comparatively steep drug prices

and the greatest number of prevalent CML cases. By

2022, GlobalData expects the market size (defined as

sales of branded therapies) to decrease to $2.119 billion

at a negative compound annual growth rate (CAGR) of

3.5%.

The major barriers responsible for the decline in sales of

CML therapeutics will include:

The launches of generic imatinib and dasatinib, and

subsequent erosion of Novartis’ Gleevec (imatinib)

and Bristol-Myers Squibb’s (BMS’) Sprycel

(dasatinib) sales

Pressure from payers for physicians to first prescribe

cheaper generic imatinib as opposed to branded

second- and third-generation tyrosine kinase

inhibitors (TKIs)

Limited uptake of pricy new market entrants, due to a

crowded market with relatively low unmet clinical

need

Drivers of growth in the CML therapeutics market will

include:

A continually growing prevalence of CML due to

improvements in overall survival rates

Increased uptake of higher-priced second-generation

TKIs in newly diagnosed patients, increasing global

sales

An increase in the branded drug treatment rate in

highly refractory or intolerant patients following the

launch of Ariad’s Iclusig (ponatinib) and Pfizer’s

Bosulif (bosutinib)

Executive Summary


The figure below illustrates the changing breakdown of

sales of CML therapeutics by region over the forecast

period.

Sales for CML by Region, 2012–2022

53%31%

16%

US 5EU Japan

2012Total: $3,013m

48%

25%

27%

US 5EU Japan

2022Total: $2,119m

Source: GlobalData

Pharmaceutical Companies Fight for Market Share in Newly Diagnosed CML Patients

The most lucrative CML segment is CP-CML patients

currently taking frontline therapy. This segment

contains the greatest number of patients, and these

patients stay on their treatment for longer than

patients who have become refractory to or intolerant

of prior therapy. Competition for patient share in this

segment is fierce, and Novartis and BMS are trying to

prove that their second-generation TKIs are superior

to Gleevec. Ariad is also aiming to penetrate this

segment.

It will follow the trend set by Novartis and BMS, as it

plans to seek a brand extension for Iclusig for newly

diagnosed patients.

Improving CML patients’ compliance with their

prescription regimens is of great commercial interest

to companies in this space. Consequently, the major

players have instituted programs and tools to

encourage patients to adhere to their prescribed

frequency of dosing.

Novartis has been the CML market leader since it

first launched Gleevec in 2001. In order to prevent a

major loss of revenue after Gleevec’s imminent

patent expiry, Novartis is fighting hard to convince

physicians to prescribe its second-generation TKI,

Tasigna, instead. However, GlobalData does not

believe Novartis’ efforts will be enough to overcome

the allure of generic imatinib. Tasigna’s long patent

life and Novartis’ marketing expertise will enable the

company to maintain its position as market leader

through 2022.

The possibility of discontinuing TKI therapy is a hot

topic in the global CML community. Novartis and

Ariad know this, and are sponsoring or collaborating

on clinical trials evaluating whether patients taking

Tasigna or Iclusig can safely discontinue therapy.

The companies hope that this strategy will help their

drugs maintain market share once they face

competition from generic imatinib.

The CML therapeutics market is now saturated with

BCR-ABL TKIs. Future players should look to

different targets in order to capture share in this

space. Novartis, BMS and Pfizer have experimented

in this area, but most projects have been

discontinued after early-phase trials.

Executive Summary


The below figure stratifies the current and future players

in the CML space by clinical and commercial strengths.

Company Portfolio Gap Analysis in CML, 2013–2022

High

Low

Clinical Attributes of Products

Low High

Hig

hLo

wCom

mer

cial

Cap

abili

ties

Source: GlobalData

The Level of Unmet Need in the CML Market is Relatively Low

The level of unmet need in the CML market is

moderately low, as most cases of CML can be

controlled by TKI therapy with minimal safety issues.

In most patients, Gleevec, Sprycel and Tasigna can

adequately prevent disease progression for several

years.

With the launch of new market entrants Bosulif,

Iclusig and Synribo for patients who are refractory to

or intolerant of Gleevec and second-generation TKIs,

there will be little room for new market entrants.

These drugs are targeted towards patients who no

longer respond to first- or second-generation TKIs,

and provide options for additional lines of therapy.

Iclusig is an oral TKI that is efficacious in patients

with the T315I BCR-ABL mutation, significantly

decreasing the level of this formerly unmet need.

The remaining unmet needs have shifted from

disease management of all CML patients to the

unique needs of small subsets of patients. Small

numbers of patients suffer from severe chronic side

effects with TKI therapy, and are not adequately

served by currently marketed therapies.

TKI therapy is not as effective with patients who have

AP or BP disease as it is in those with CP CML. This

small fraction of patients has a much higher level of

unmet need than those in chronic phase.

Environmental unmet needs also affect the CML

market. Key opinion leaders have emphasized that

the cost of therapy places a major financial burden

on CML patients and global healthcare systems, a

burden that will be even more difficult to bear as the

global prevalence increases. In markets like the US,

significant cost-sharing can reduce compliance rates

of

The unusually poor compliance of CML patients with

their oral TKI therapy is often discussed in the

secondary literature and by key opinion leaders.

There is little agreement on how low compliance

rates actually are, but it is clear that many patients do

not take their medication exactly as prescribed. This

leads to higher rates of relapse in patients, and less

than optimal sales of TKIs.

Executive Summary


The CML Space is Crowded with Safe and Effective BCR-ABL TKIs, Leaving Little Room for New Market Entrants

In order to be commercially viable, new therapies

hoping to demand premium pricing must seek to

improve on both the safety and efficacy of currently

marketed drugs, particularly in patients with

advanced disease, or completely eradicate the

disease so that a patient can discontinue therapy.

Neither of these criteria will be easy to meet. While

today’s drugs are highly effective, it is important to

note that there is incongruence between patients’

and physicians’ assessment of the side effects

profiles of currently marketed TKIs, and how those

side effects impact patients’ quality of life. Future

market entrants must be certain to address this need,

especially if they hope to penetrate the newly

diagnosed patient segment.

Meeting the clinical unmet needs of good efficacy

and safety are paramount for any oncology

indication. However, the CML market now contains

multiple safe and effective treatment options, and the

prevalence of CML is expected to continue to

increase as patients live longer. GlobalData expects

the cost of therapy to become an increasing factor in

physicians’ prescribing choices, catalyzed by the

entry of generics. Potential new market entrants must

be willing to compete on price if they want to capture

maximum patient share in this market.

The Pipeline for CML is Weak Due to the Low Levels of Unmet Needs

The stringent criteria for acceptable safety and

efficacy profiles of BCR-ABL TKIs make CML a high-

risk area for pharmaceutical research and

development.

At this time, there are no late-stage drugs in

development for CML, and very few early-stage

projects. Pharmaceutical companies have been quick

to discontinue the development of CML therapies,

often in light of safety or side effects concerns.

Early-stage pipeline products are first-in-class,

targeting new signaling pathways and proteins other

than BCR-ABL. If successful, they will likely be used

in combination with currently marketed TKIs to treat

patients with advanced phase (AP) or blast phase

(BP) CML.

What Do the Physicians Think?

Our experts believe that the most promising new market

entrant is Ariad’s Iclusig (ponatinib). Although it is highly

efficacious, they expect its use to be limited to later lines

of therapy as a result of its premium price and associated

toxicities.

“Ponatinib is a really good drug and I think that it’s the

one that has a chance to really move up. Because I think

a lot of [physicians], they are thinking about it for people

who have T315I mutations…but it works on almost all the

mutations. So I think there is going to be a move to kind

of use that as a salvage therapy, especially for people

who have started on dasatinib and nilotinib and don’t do

well. They will go to ponatinib.”

US Key Opinion Leader, February 2013

Executive Summary


“Ponatinib is a very active drug against all mutations, and

it has a very, very good chance for third-line, of course,

but also [for] second-line treatment. The study for first-

line treatment is ongoing, that means I expect some more

[use] in first-line as well. But then, having four drugs

available for first-line treatment…it really depends on the

economics.”

5EU Key Opinion Leader, February 2013

Key opinion leaders in the 7MM believe that the safety

and cost will be the most critical factors influencing future

prescribing patterns.

“In the next five years, the most important factors will be

the side effects and the cost [of a drug] more than the

efficacy, because all these drugs are very effective.”

5EU Key Opinion Leader, January 2013

Physicians are eager for the launch of generic imatinib.

They believe that although other therapies are stronger,

the cost savings associated with generic imatinib will

make it the drug of choice for low- to intermediate-risk

newly diagnosed CP-CML patients. In many markets,

particularly in the 5EU, they expect the use of generic

imatinib to be mandated by payers.

“I think that when you calculate the prevalence of CML in

the world, I think that [using generic imatinib in newly

diagnosed CML patients] is the right decision. You can

help more patients when you use the cheaper drug, in

total. But of course, the individual patient would benefit

from the more expensive and efficacious drug; but the

worldwide population of CML patients will benefit from

the cheaper drug because more patients can be treated

with the cheaper drug. That’s the responsibility we all

have.”

5EU Key Opinion Leader, February 2013

The discontinuation of TKI therapy is the future of CML,

and something highly desired by patients.

“The target for the 21st century must be to stop a tyrosine

kinase inhibitor in a way that a person doesn’t need to

take it for the rest of their life. So all attention focuses on

a) how you can increase the proportion of patients who

achieve a complete, durable, molecular response and b)

what additional measures you can take to ensure that

eventually a TKI can be stopped.”

5EU Key Opinion Leader, January 2013

Currently, there is no “best” sequence of TKIs to

prescribe for CML patients. More long-term follow-up

data is needed to justify routinely prescribing second- or

third-generation TKIs for newly diagnosed CP-CML

patients rather than Gleevec.

“There is still not enough data for us to recommend any

specific treatment other than just ‘tyrosine kinase

inhibitors’ for upfront therapy for CML. Especially

regarding an increase in survival.”

US Key Opinion Leader, February 2013

Table of Contents


1 Table of Contents

1 Table of Contents ............................................................................................................... 7

1.1 List of Tables ............................................................................................................. 14

1.2 List of Figures ........................................................................................................... 17

2 Introduction ....................................................................................................................... 18

2.1 Catalyst ..................................................................................................................... 18

2.2 Related Reports ........................................................................................................ 18

2.3 Upcoming Related Reports ........................................................................................ 18

3 Disease Overview ............................................................................................................. 19

3.1 Etiology and Pathophysiology .................................................................................... 19

3.1.1 Etiology ............................................................................................................... 19

3.1.2 Pathophysiology.................................................................................................. 20

3.1.3 Clinical Staging ................................................................................................... 22

3.1.4 Prognosis ............................................................................................................ 23

3.1.5 Quality of Life ...................................................................................................... 24

3.2 Symptoms ................................................................................................................. 24

4 Epidemiology .................................................................................................................... 26

4.1 Risk Factors and Co-morbidities ................................................................................ 26

4.1.1 Exposure to ionizing radiation may not lead to the development of CML .............. 26

4.1.2 Obesity and weight gain in adulthood play important roles in CML risk................. 27

4.2 Global and Historical Trends ..................................................................................... 27

4.2.1 Incidence ............................................................................................................ 27

4.2.2 Prevalence and Survival ...................................................................................... 29

4.2.3 Mortality .............................................................................................................. 29

4.3 Forecast Methodology ............................................................................................... 30

4.3.1 Sources Used ..................................................................................................... 31

4.3.2 Forecast Assumptions and Methods, Incident Cases ........................................... 33

4.3.3 Forecast Assumptions and Methods, Prevalent Cases ........................................ 35

Table of Contents


4.3.4 Sources Not Used ............................................................................................... 35

4.4 Epidemiological Forecast of Chronic Myeloid Leukemia (2012–2022) ........................ 36

4.4.1 Incident Cases of Chronic Myeloid Leukemia ...................................................... 36

4.4.2 Incident Cases of Chronic Myeloid Leukemia by Age Group ................................ 38

4.4.3 Incident Cases of Chronic Myeloid Leukemia by Sex ........................................... 39

4.4.4 Age-Standardized Incidence Rates of Chronic Myeloid Leukemia ........................ 40

4.4.5 Incident Cases of Chronic Myeloid Leukemia with Ph+ ........................................ 41

4.4.6 Incident Cases of Chronic Myeloid Leukemia by Phase at Diagnosis ................... 42

4.4.7 Prevalent Cases of Chronic Myeloid Leukemia .................................................... 43

4.5 Discussion................................................................................................................. 47

4.5.1 Epidemiological Forecast Insight ......................................................................... 47

4.5.2 Limitations of the Analysis ................................................................................... 48

4.5.3 Strengths of the Analysis ..................................................................................... 49

5 Disease Management ....................................................................................................... 50

5.1 Global Trends ........................................................................................................... 50

5.1.1 Treatment Overview ............................................................................................ 50

5.1.2 Diagnostic Tests ................................................................................................. 52

5.1.3 Genetic Testing ................................................................................................... 53

5.1.4 Monitoring Patient Response to Treatment .......................................................... 55

5.1.5 Future Directions: Discontinuation Therapy ......................................................... 58

5.2 US ........................................................................................................................... 59

5.2.1 Diagnosis and Monitoring .................................................................................... 59

5.2.2 Clinical Practice .................................................................................................. 60

5.2.3 Genetic Testing ................................................................................................... 61

5.3 France ...................................................................................................................... 62



5.3.3 Genetic Testing ................................................................................................... 63

Table of Contents


5.4 Germany ................................................................................................................... 64



5.4.3 Genetic Testing ................................................................................................... 66

5.5 Italy ........................................................................................................................... 66



5.5.3 Genetic Testing ................................................................................................... 68

5.6 Spain ........................................................................................................................ 68


5.6.2 Clinical Treatment ............................................................................................... 68

5.6.3 Genetic Testing ................................................................................................... 70

5.7 UK ........................................................................................................................... 70



5.7.3 Genetic Testing ................................................................................................... 72

5.8 Japan ........................................................................................................................ 73



5.8.3 Genetic Testing ................................................................................................... 74

6 Competitive Assessment ................................................................................................... 75

6.1 Overview ................................................................................................................... 75

6.2 Strategic Competitor Assessment .............................................................................. 75

6.3 Product Profiles- Major Brands .................................................................................. 78

6.3.1 Gleevec (imatinib) ............................................................................................... 78

6.3.2 Sprycel (dasatinib) .............................................................................................. 83

6.3.3 Tasigna (nilotinib) ................................................................................................ 90

6.3.4 Bosulif (bosutinib) ............................................................................................... 96

Table of Contents


6.3.5 Iclusig (ponatinib) .............................................................................................. 102

6.3.6 Synribo (omacetaxine mepesuccinate) .............................................................. 107

6.3.7 Minor Therapeutic Classes ................................................................................ 112

7 Opportunity and Unmet Need .......................................................................................... 114

7.1 Overview ................................................................................................................. 114

7.2 Unmet Need: A Drug that Can Cure CML ................................................................ 115

7.3 Unmet Need: Lower Annual Cost of Therapy ........................................................... 116

7.4 Unmet Need: Treatments for Patients Who Have Primary Resistance to or are

Refractory to TKIs ................................................................................................... 117

7.5 Unmet Need: More Efficacious Treatments for AP and BP CML .............................. 117

7.6 Unmet Need: Therapies with Fewer Chronic Side Effects ........................................ 118

7.7 Unmet Need: Better Compliance from Patients on Long-Term Oral Therapy ............ 118

7.8 Unmet Need: Methods of Determining the Optimal Therapy for a Patient ................. 119

7.9 Opportunity: Exploration into Discontinuation Therapy ............................................. 119

7.10 Opportunity: Companion Devices to Enhance Patient Adherence to Oral Therapy ... 120

7.11 Opportunity: Therapies with BCR-ABL Independent MOAs ...................................... 120

7.12 Opportunity: Extended-Release Formulations of TKIs .............................................. 121

7.13 Opportunity: Biomarkers to Identify the Optimal Therapy for a Given Patient ............ 121

8 Pipeline Assessment....................................................................................................... 122

8.1 Overview ................................................................................................................. 122

8.2 Clinical Trial Mapping .............................................................................................. 123

8.2.1 Clinical Trials by Country ................................................................................... 123

8.3 Clinical Trials by Phase and Trial Status .................................................................. 124

8.4 Innovative Early-Stage Approaches ......................................................................... 125

8.4.1 Project 1: The Wnt Signaling Pathway ............................................................... 126

8.4.2 Project 2: Jak2 Inhibitors ................................................................................... 127

8.4.3 Project 3: Grb-2................................................................................................. 128

8.4.4 Case Study: Smoothened Inhibitors .................................................................. 128

9 Current and Future Players ............................................................................................. 130

Table of Contents


9.1 Overview ................................................................................................................. 130

9.2 Trends in Corporate Strategy................................................................................... 132

9.3 Company Profiles .................................................................................................... 133

9.3.1 Novartis ............................................................................................................ 133

9.3.2 BMS.................................................................................................................. 136

9.3.3 Ariad ................................................................................................................. 138

9.3.4 Pfizer ................................................................................................................ 141

10 Market Outlook ............................................................................................................... 144

10.1 Global Markets ........................................................................................................ 144

10.1.1 Forecast............................................................................................................ 144

10.1.2 Drivers and Barriers – Global Issues ................................................................. 146

10.2 US ......................................................................................................................... 149

10.2.1 Forecast............................................................................................................ 149

10.2.2 Key Events ....................................................................................................... 152

10.2.3 Drivers and Barriers .......................................................................................... 152

10.3 France .................................................................................................................... 155

10.3.1 Forecast............................................................................................................ 155

10.3.2 Key Events ....................................................................................................... 158


10.4 Germany ................................................................................................................. 160

10.4.1 Forecast............................................................................................................ 161

10.4.2 Key Events ....................................................................................................... 162


10.5 Italy ......................................................................................................................... 164

10.5.1 Forecast............................................................................................................ 165

10.5.2 Key Events ....................................................................................................... 166


10.6 Spain ...................................................................................................................... 168

Table of Contents


10.6.1 Forecast............................................................................................................ 169

10.6.2 Key Events ....................................................................................................... 170


10.7 UK ......................................................................................................................... 172

10.7.1 Forecast............................................................................................................ 174

10.7.2 Key Events ....................................................................................................... 175


10.8 Japan ...................................................................................................................... 178

10.8.1 Forecast............................................................................................................ 179

10.8.2 Key Events ....................................................................................................... 181


11 Appendix ........................................................................................................................ 184

11.1 Bibliography ............................................................................................................ 184

11.2 Abbreviations .......................................................................................................... 195

11.3 Methodology ........................................................................................................... 197

11.4 Forecasting Methodology ........................................................................................ 197

11.4.1 Diagnosed CML patients ................................................................................... 197

11.4.2 Drug-treated Patients on X Line of Therapy ....................................................... 198

11.4.3 Drugs Included in Each Therapeutic Class ........................................................ 198

11.4.4 Launch and Patent Expiry Dates ....................................................................... 198

11.4.5 General Pricing Assumptions ............................................................................ 199

11.4.6 Compliance Assumptions for Oral TKIs ............................................................. 201

11.4.7 Individual Drug Assumptions ............................................................................. 202

11.4.8 Generic Erosion ................................................................................................ 205

11.4.9 Pricing of New Market Entrants ......................................................................... 205

11.5 Physicians and Specialists Included in this Study .................................................... 206

11.6 Survey of High Prescribing Physicians ..................................................................... 207

11.7 About the Authors ................................................................................................... 208

Table of Contents


11.7.1 Authors ............................................................................................................. 208

11.7.2 Epidemiologists ................................................................................................. 209

11.7.3 Global Director of Epidemiology and Clinical Trials Analysis .............................. 210

11.7.4 Global Head of Healthcare ................................................................................ 211

11.8 About GlobalData .................................................................................................... 212

11.9 Contact Us .............................................................................................................. 212

11.10 Disclaimer ......................................................................................................... 212

Table of Contents


1.1 List of Tables

Table 1: The Staging of CML as Defined by Commonly Used Staging Systems ............... 22

Table 2: Prognostic Scoring Systems for CML ................................................................. 23

Table 3: Common Symptoms of CML by Disease Phase ................................................. 25

Table 4: Reported Incidence of Chronic Myeloid Leukemia for the US, EU, and Japan .... 28

Table 5: US and Japan, Annual Percentage Change (APC) in CML Mortality Rates, by

Sex, All Ages, %, 1993–2008 ............................................................................ 30

Table 6: 7MM, Sources of CML Incidence and Prevalence Data ...................................... 31

Table 7: 7MM, Incident Cases of Chronic Myeloid Leukemia, All Ages, Men and Women,

N, 2012–2022 .................................................................................................... 37

Table 8: 7MM, Incident Cases of CML, by Age Group, Men and Women, N, 2012 ........... 38

Table 9: 7MM, Incident Cases of CML, by Sex, All Ages, N (Row %), 2012 ..................... 39

Table 10: 7MM, Incident Cases of CML, by Ph+, Men and Women, All Ages, N, 2012 ....... 41

Table 11: 7MM, Incident Cases of CML, by Phase at Diagnosis, Men and Women, All Ages,

N, 2012 ............................................................................................................. 42

Table 12: 7MM, Five- and 10-Year Prevalent Cases of Chronic Myeloid Leukemia, All Ages,

Men and Women, N, 2012–2022 ....................................................................... 45

Table 13: Most Commonly Followed Treatment Guidelines for CML .................................. 51

Table 14: Most Prescribed First-Line Therapies for CP, AP and BP CML in the Global

Markets, 2013 ................................................................................................... 52

Table 15: Suggested Treatments for CML Patients with Selected BCR-ABL Kinase Domain

Mutations .......................................................................................................... 55

Table 16: CML Response Types, Criteria, and Corresponding Tests ................................. 56

Table 17: Leading Treatments for Chronic Myeloid Leukemia, 2013 .................................. 77

Table 18: Product Profile – Gleevec .................................................................................. 79

Table 19: Hematologic and Cytogenetic Reponses to Gleevec in Newly Diagnosed CML

Patients ............................................................................................................. 81

Table 20: Gleevec SWOT Analysis, 2013 .......................................................................... 82

Table 21: Global Sales Forecasts ($m) for Gleevec in CML, 2012–2022 ........................... 83

Table 22: Product Profile – Sprycel ................................................................................... 85

Table of Contents


Table 23: Hematologic and Cytogenetic Reponses to Sprycel in Imatinib Resistant or

Intolerant Advanced Phase CML ....................................................................... 86

Table 24: Sprycel SWOT Analysis, 2013 ........................................................................... 88

Table 25: Global Sales Forecasts ($m) for Sprycel in CML, 2012–2022 ............................. 90

Table 26: Product Profile – Tasigna .................................................................................. 91

Table 27: Molecular and Cytogenetic Responses of Tasigna Compared with Gleevec in

Newly Diagnosed Ph+ CML in CP ..................................................................... 92

Table 28: Tasigna SWOT Analysis, 2013 .......................................................................... 94

Table 29: Global Sales Forecasts ($m) for Tasigna in CML, 2012–2022 ............................ 95

Table 30: Product Profile – Bosulif .................................................................................... 97

Table 31: Bosulif SWOT Analysis, 2013 .......................................................................... 100

Table 32: Global Sales Forecasts ($m) for Bosulif in CML, 2012–2022 ............................ 101

Table 33: Product Profile – Iclusig ................................................................................... 103

Table 34: Iclusig SWOT Analysis, 2013 ........................................................................... 106

Table 35: Global Sales Forecasts ($m) for Iclusig in CML, 2012–2022 ............................ 107

Table 36: Product Profile – Synribo ................................................................................. 109

Table 37: Synribo SWOT Analysis, 2013 ......................................................................... 111

Table 38: Global Sales Forecasts ($m) for Synribo in CML, 2012–2022 .......................... 112

Table 39: Summary of Minor Therapeutic Classes, 2013 ................................................. 113

Table 40: Overall Unmet Needs – Current Level of Attainment ........................................ 115

Table 41: CML – Clinical Trials by Phase and Status, 2013 ............................................. 124

Table 42: Early-stage Pipeline Projects in CML ............................................................... 126

Table 43: Key Companies in the CML Market, 2013 ........................................................ 131

Table 44: Novartis’ CML Portfolio Assessment, 2013 ...................................................... 135

Table 45: Novartis SWOT Analysis, 2013 ........................................................................ 135

Table 46: BMS’ CML Portfolio Assessment, 2013 ............................................................ 137

Table 47: BMS SWOT Analysis, 2013 ............................................................................. 138

Table 48: Ariad’s CML Portfolio Assessment, 2013 ......................................................... 140

Table 49: Ariad SWOT Analysis, 2013 ............................................................................ 141

Table of Contents


Table 50: Pfizer’s CML Portfolio Assessment, 2013......................................................... 142

Table 51: Pfizer SWOT Analysis, 2013 ............................................................................ 143

Table 52: Global Sales Forecasts ($m) for CML, 2012–2022 ........................................... 145

Table 53: Chronic Myeloid Leukemia Market – Drivers and Barriers, 2013....................... 146

Table 54: Global Sales Forecasts ($m) for CML in the US, 2012–2022............................ 150

Table 55: Key Events Impacting Sales of CML Therapeutics in the United States, 2013 .. 152

Table 56: CML Market in the United States – Drivers and Barriers, 2013 ......................... 152

Table 57: Sales Forecasts ($m) for CML Therapeutics in France, 2012–2022 ................. 156

Table 58: Key Events Impacting Sales of CML Therapeutics in France, 2013 .................. 158

Table 59: CML Market in France – Drivers and Barriers, 2013 ......................................... 158

Table 60: Sales Forecasts ($m) for CML Therapeutics in Germany, 2012–2022 .............. 161

Table 61: Key Events Impacting Sales of CML Therapeutics in Germany, 2013 .............. 162

Table 62: CML Market in Germany – Drivers and Barriers, 2013 ..................................... 162

Table 63: Sales Forecasts ($m) for CML Therapeutics in Italy, 2012–2022 ...................... 165

Table 64: Key Events Impacting Sales of CML Therapeutics in Italy, 2013 ...................... 166

Table 65: CML Market in Italy – Drivers and Barriers, 2013 ............................................. 166

Table 66: Sales Forecasts ($m) for CML Therapeutics in Spain, 2012–2022 ................... 169

Table 67: Key Events Impacting Sales of CML Therapeutics in Spain, 2013 .................... 170

Table 68: CML Market in Spain – Drivers and Barriers, 2013 ........................................... 170

Table 69: Sales Forecasts ($m) for CML Therapeutics in the United Kingdom, 2012–202 174

Table 70: Key Events Impacting Sales of CML Therapeutics in the UK, 2013 .................. 175

Table 71: CML Market in the UK – Drivers and Barriers, 2013 ......................................... 175

Table 72: Sales Forecasts ($m) for CML Therapeutics in Japan, 2012–2022 ................... 179

Table 73: Key Events Impacting Sales of CML Therapeutics in Japan, 2013 ................... 181

Table 74: CML Market in Japan – Drivers and Barriers, 2013 .......................................... 181

Table 75: Key Launch Dates ........................................................................................... 198

Table 76: Key Patent Expiries ......................................................................................... 198

Table 77: Physicians Surveyed, by Country..................................................................... 207

Table of Contents


1.2 List of Figures

Figure 1: Translocation of Chromosomes 9 and 22 ........................................................... 20

Figure 2: Comparison of Normal and Leukemia Blood Cells ............................................. 21

Figure 3: 7MM, Incident Cases of Chronic Myeloid Leukemia, All Ages, Men and Women,

N, 2012–2022 .................................................................................................... 37

Figure 4: 7MM, Incident Cases of Chronic Myeloid Leukemia, by Age Group, Men and

Women, N, 2012 ............................................................................................... 39

Figure 5: 7MM, Incident Cases of Chronic Myeloid Leukemia, All Ages, By Sex , N, 2012 40

Figure 6: 7MM, Age-Standardized Incidence of Chronic Myeloid Leukemia, All Ages, Men

and Women, Cases per 100,000 People, 2012 .................................................. 41

Figure 7: 7MM, Incident Cases of Chronic Myeloid Leukemia, by Phase at Diagnosis, All

Ages, Men and Women, N, 2012 ....................................................................... 43

Figure 8: 7MM, Five-Year Prevalent Cases of Chronic Myeloid Leukemia, All Ages, Men

and Women, N, 2012–2022 ............................................................................... 46

Figure 9: 7MM, 10-Year Prevalent Cases of Chronic Myeloid Leukemia, All Ages, Men and

Women, N, 2012–2022 ...................................................................................... 46

Figure 10: CML Therapeutics – Clinical Trials by Country, 2013 ....................................... 123

Figure 11: Company Portfolio Gap Analysis in CML, 2013–2022 ...................................... 131

Figure 12: Global Sales for CML Therapeutics by Region, 2012–2022 ............................. 145

Figure 13: Sales for CML Therapeutics in the United States by Brand, 2012–2022 ........... 151

Figure 14: Sales for CML in France by Brand, 2012–2022 ................................................ 157

Figure 15: Sales for CML Therapeutics in Germany by Brand, 2012–2022 ....................... 161

Figure 16: Sales for CML in Italy by Brand, 2012–2022 .................................................... 165

Figure 17: Sales for CML Therapeutics in Spain by Brand, 2012–2022 ............................. 169

Figure 18: Sales for CML Therapeutics in the UK by Brand, 2012–2022 ........................... 174

Figure 19: Sales for CML Therapeutics in Japan by Brand, 2012–2022 ............................ 180

Introduction


2 Introduction

2.1 Catalyst

The launch of Novartis’ BCR-ABL inhibitor Gleevec (imatinib) in 2001 forever changed the

treatment of chronic myeloid leukemia (CML). With Gleevec, and later the second-generation TKIs,

BMS’ Sprycel (dasatinib) and Novartis’ Tasigna (nilotinib), CML has become less of a death

sentence and more of a chronic, manageable condition. The CML market has grown as a result of

this steadily increasing disease prevalence, and the high cost of branded TKIs places a heavy

financial burden on patients and global healthcare systems.

The effects of this burden will have a major impact on the future CML market. The sustained and

escalating costs of branded TKI therapy have left payers, physicians and patients anxiously

awaiting the launch of generic imatinib. Swift erosion of Gleevec sales will follow, and physicians

will be left with the question of whether to prescribe second-generation TKIs for newly diagnosed

patients, or the more cost-effective generic Gleevec. Ultimately, the launch and uptake of generic

imatinib will be the primary driver of the decreasing size of the global CML market. New entrants

Pfizer’s Bosulif (bosutinib) and Ariad’s Iclusig (ponatinib) will be welcome treatment options for

patients who are refractory to or intolerant of Gleevec, Sprycel and Tasigna. In light of the

aforementioned fiscal constraints, these drugs will be predominantly prescribed in later lines of

therapy, restricting their ability to compensate for the market’s loss of Gleevec sales.

2.2 Related Reports

GlobalData (2012) Prostate Cancer – Global Drug Forecast and Market Analysis to 2022:

Event-Driven Update, December 2012, GDHC29PIDR.

2.3 Upcoming Related Reports

GlobalData (2013). Non-Small Cell Lung Cancer - Global Drug Forecast and Market Analysis

to 2022, May 2013.

GlobalData (2013). Colorectal Cancer – Global Drug Forecast and Market Analysis to 2022,

May 2013.

Appendix


11.8 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, Boston, London, India and Singapore.

11.10 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.

Documents

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