Stakeholder Opinions: Myelodysplastic Syndromes - First approvals spur some interest in a niche market

Published by: Datamonitor

Published: Dec. 19, 2007 - 143 Pages

Table of Contents

ABOUT DATAMONITOR HEALTHCARE

About the Oncology pharmaceutical analysis team

CHAPTER 1 EXECUTIVE SUMMARY

Scope of analysis

Datamonitor insight into the MDS market

Related reports

Upcoming reports

CHAPTER 2 OVERVIEW OF MYELODYSPLASTIC SYNDROMES

Introduction

Myelodysplastic syndromes (MDS) are a heterogeneous group of hematological malignancies

Risk factors in the development of MDS

Hallmark of MDS - A hypercellular bone marrow with dysplastic changes in combination with peripheral cytopenias

Hypercellularity

Dysplasia

Peripheral cytopenias

Mortality often results from the complications of peripheral blood cytopenias

MDS is often a diagnosis of exclusion

The molecular pathogenesis of MDS

Chromosomal abnormalities are common but little is known about the molecular basis of the disease

MDS arises as a multistep process

Accelerated apoptosis underlies the cytopenias in early MDS

Excessive proliferation accompanies disease progression

The classification of MDS

French American British (FAB) System

The FAB classification system is based on morphological criteria

Limitations of the FAB system

World Health Organization (WHO) System

The WHO classification system incorporates new diagnostic information

Classification of chronic myelomonocytic leukemia (CMML)

Limitations of the WHO classification system

International Prognostic Scoring System (IPSS)

The IPSS classification system is the current standard for evaluating prognosis in MDS patients

Limitations of the IPSS system

Epidemiology

The epidemiology of MDS is poorly monitored

MDS is predominantly a disease of the elderly

A recent study shows that the majority of patients fall into the IPSS Low and Intermediate-1 risk groups

The incidence of MDS is expected to rise

There will be more than 57,000 new cases of MDS across the seven major markets in 2016

CHAPTER 3 CURRENT TREATMENT OPTIONS FOR MDS

Overview of MDS management

The aim of treatment differs in lower- and higher-risk patients

The choice of therapy is not solely dependent on a patient's IPSS risk group

Summary of current trends in MDS management

The management of IPSS lower-risk MDS patients

Supportive care approaches

Red blood cell (RBC) transfusion and iron chelation therapy

Erythropoietin (EPO)

Granulocyte-colony stimulating factor (G-CSF)

Platelet transfusions

Non-chemotherapy low-intensity agents

Thalomid (thalidomide; Celgene/Pharmion)

Revlimid (lenalidomide; Celgene)

Antithymocyte globulin and cyclosporin A

Anti-TNF therapy

Vitamin D analogs

Low-intensity chemotherapy

Vidaza and Dacogen

The management of IPSS higher-risk patients

Low-intensity chemotherapy - the hypomethylating agents

Vidaza (azacitidine; Pharmion)

Dacogen (decitabine; MGI Pharma)

Comparisons between Vidaza and Dacogen

Hematopoietic stem cell transplantation

HSCT is the only potentially curative option for MDS patients but its use in older patients is problematic

The morbidity and mortality risks associated with HSCT often prohibits its use in lower-risk patients

Different transplantation options exist for the treatment of MDS

High-intensity chemotherapy

High-intensity chemotherapy is an alternative option for higher-risk patients

The use of high-intensity chemotherapy may be difficult to justify

The management of CMML

Supportive care and low-intensity therapy

High-intensity therapy

CHAPTER 4 UNMET NEEDS IN MDS

Summary of unmet needs

A better understanding of the molecular pathogenesis

Increased R&D efforts

Low-intensity therapies with improved efficacy and better toxicity profiles

Low-intensity therapies to improve symptoms of anemia and quality of life

More effective management of thrombocytopenia

Improved HSCT procedures

CHAPTER 5 PIPELINE ANALYSIS

Summary

Pipeline overview

Late-phase product pipeline for MDS

Phase II product pipeline for MDS

Phase I product pipeline for MDS

Late-phase MDS drug profiles

Sarasar (Lonafarnib; Schering-Plough)

Drug overview

Key historical events

Clinical trial data

Datamonitor comments

Phase II MDS drug profiles

MGCD0103 (Pharmion/MethylGene)

Drug overview

Key historical events

Clinical trial data

Romiplostim (Amgen)

Drug overview

Key historical events

Clinical trial data

Ceflatonin (Myelostat; ChemGenex Pharmaceuticals)

Drug overview

Key historical events

Clinical trial data

Zarnestra (Tipifarnib; Johnson & Johnson)

Drug overview

Key historical events

Clinical trial data

Cloretazine (VNP40101M; Vion Pharmaceuticals)

Drug overview

Key historical events

Clinical trial data

Clolar/Evoltra (Clofarabine; Genzyme)

Drug overview

Key historical events

Clinical trial data

Telintra (TLK-199; Telik)

Drug overview

Key historical events

Clinical trial data

APPENDIX

Bibliography

Journals

Websites

Other

Key opinion leaders

List of tables

List of figures

Abbreviations

About Datamonitor

About Datamonitor Healthcare

About the Oncology analysis team

Disclaimer

List of Tables

Table 1: Myelodysplastic syndromes (MDS)

Table 2: Common recurring chromosomal abnormalities in MDS

Table 3: French American British (FAB) classification of MDS

Table 4: World Health Organization (WHO) classification of MDS

Table 5: WHO classification of myelodysplastic/myeloproliferative diseases (MDS/MPD)

Table 6: Survival and progression to AML for MDS patients within the IPSS risk groups

Table 7: IPSS classification of MDS patients

Table 8: MDS incidence in the seven major markets, 2002-2016

Table 9: Treatment guidelines for MDS

Table 10: Iron chelators available in the US and EU markets, 2007

Table 11: Branded erythropoietins available in the US and EU markets, 2007

Table 12: Erythropoietins: ongoing clinical trials in MDS, 2007

Table 13: Branded colony-stimulating factors available in the US and EU, 2007

Table 14: Thalomid: key historical events

Table 15: Thalomid as a single agent in MDS: clinical trial results

Table 16: Revlimid: key historical events

Table 17: Revlimid: ongoing clinical trials in MDS, 2007

Table 18: Antithymocyte globulin (ATG) available in the US and EU, 2007

Table 19: Antithymocyte globulin: ongoing clinical trials in MDS, 2007

Table 20: Vidaza: Key historical events

Table 21: Vidaza: ongoing clinical trials in MDS, 2007

Table 22: Response criteria used in the Phase III study of Vidaza versus supportive care in MDS patients

Table 23: Dacogen: key historical events

Table 24: Dacogen: ongoing clinical trials in MDS, 2007

Table 25: Response criteria used in the Phase III study of Dacogen versus supportive care in MDS patients

Table 26: Types of hematopoietic stem cell transplantation (HSCT)

Table 27: Comparative results of RIC HSCT studies

Table 28: High-intensity chemotherapy regimens in MDS: clinical trial results

Table 29: Pipeline drugs in Phase III development for MDS, 2007

Table 30: Pipeline drugs in Phase II development for MDS, 2007

Table 31: Pipeline drugs in Phase I development for MDS, 2007

Table 32: Sarasar: key historical events

Table 33: Ongoing clinical trials involving Sarasar in MDS, 2007

Table 34: MGCD0103: key historical events

Table 35: Ongoing clinical trials for MGCD0103 in MDS, 2007

Table 36: Romiplostim: key historical events

Table 37: Ongoing clinical trials for Romiplostim in MDS, 2007

Table 38: Ceflatonin: Key historical events

Table 39: Zarnestra: Key historical events

Table 40: Ongoing clinical trials involving Zarnestra in MDS, 2007

Table 41: Final results from a Phase II study of Zarnestra in Intermediate- and High-risk MDS

Table 42: Cloretazine: Key historical events

Table 43: Ongoing clinical trials involving Cloretazine in MDS, 2007

Table 44: Clolar: Key historical events

Table 45: Ongoing clinical trials involving Clolar in MDS, 2007

Table 46: Telintra: key historical events

Table 47: Ongoing clinical trials involving Telintra in MDS, 2007

Table 48: Abbreviations used in Stakeholder Opinions: Myelodysplastic Syndromes

List of Figures

Figure 1: Risk factors implicated in the onset of MDS

Figure 2: The hematopoiesis process

Figure 3: Hypercellularity and peripheral blood cytopenias in MDS

Figure 4: The pathophysiology of MDS

Figure 5: Median survival for each WHO MDS subtype

Figure 6: Cumulative risk of progressing to AML after 2 years for each MDS WHO subtype

Figure 7: International Prognostic Scoring System (IPSS) for MDS

Figure 8: Male and female incidence of MDS in the US over and under 65 years of age as a percentage of the total US patient population, 2007

Figure 9: MDS incidence in the seven major markets, 2007 and 2016

Figure 10: Incidence of MDS subtypes in the seven major markets, 2007

Figure 11: Factors complicating the management of MDS

Figure 12: Summary of supportive care, low-intensity and high-intensity therapies available for the management of MDS

Figure 13: Summary of current trends in the management of lower-risk MDS

Figure 14: Summary of current trends in the management of higher-risk MDS

Figure 15: NCCN treatment guidelines for lower-risk MDS patients (IPSS Low/Intermediate-1)

Figure 16: The implications of long-term transfusion dependence

Figure 17: Thrombocytopenia in MDS

Figure 18: Phase II study of Revlimid in MDS patients with del(5q)

Figure 19: Phase II study of Revlimid in MDS patients without del(5q)

Figure 20: Phase II study of Revlimid in Intermediate-2/High-risk MDS with del(5q): Interim results

Figure 21: Phase III study of Lymphoglobuline with cyclosporine versus best supportive care in Low/Intermediate-1 MDS

Figure 22: NCCN treatment guidelines for IPSS Intermediate-2/High-risk MDS patients

Figure 23: Phase III study of Vidaza versus supportive care in MDS patients

Figure 24: Phase III study of Vidaza versus conventional care regimens in higher-risk MDS

Figure 25: Phase III study of Dacogen versus supportive care in patients with MDS

Figure 26: US sales of Vidaza and Dacogen, Q1 2006-Q3 2007

Figure 27: Summary of unmet needs in the treatment of MDS

Figure 28: Clinical development pipeline for MDS, 2007

Figure 29: Proteins undergoing farnesylation by farnesyltransferase

Figure 30: Phase I/II study of Sarasar in advanced MDS and CMML

Figure 31: Phase I/II study of Romiplostim in Low-risk/Intermediate-1 MDS

Figure 32: Proteins undergoing farnesylation by farnesyltransferase

Figure 33: Phase II study of Zarnestra in Intermediate and High-risk MDS

Figure 34: Phase II study of Cloretazine in AML and high-risk MDS

Figure 35: Phase II studies of intravenous and oral Clolar in higher-risk MDS

Figure 36: Phase I/II study of intravenous Telintra (liposomes for injection) in MDS

Abstract

About MarketResearch.com
MarketResearch.com is an online aggregator selling over 160,000 market research reports, company profiles and country profiles from over 600 research firms. Our reports will provide you with the critical business and competitive intelligence you need for strategic planning and marketing research. Coverage includes the US, UK, Europe, Asia and global markets.