Table of Contents
- CHAPTER ONE: INTRODUCTION
-
- STUDY GOALS AND OBJECTIVES
- REASONS FOR DOING THE STUDY
- SCOPE OF REPORT
- METHODOLOGY
- ANALYST CREDENTIALS
- RELATED BCC STUDIES
- BCC ONLINE SERVICES
- DISCLAIMER
-
- CHAPTER TWO: SUMMARY
-
- GENETIC DISORDER MARKETS: MAJOR MARKETS SUMMARY
- SUMMARY TABLE A GLOBAL GENETIC DISORDER PRODUCT REVENUES BY MAJOR MARKET SEGMENT, THROUGH 2014 ($ MILLIONS)
- SUMMARY FIGURE GLOBAL GENETIC DISORDER PRODUCT REVENUES, 2007-2014 ($ MILLIONS)
LEADING MANUFACTURERS
- SUMMARY TABLE B TOTAL COMPANY SALES FOR GENETIC DISORDER TREATMENT, 2009 ($ MILLIONS)
CHAPTER THREE: INDUSTRY OVERVIEW
INTRODUCTION
HISTORY OF GENETIC RESEARCH
OVERVIEW OF DNA AND GENES
THE HUMAN GENOME PROJECT
HAPMAP PROJECT
TERMINOLOGY
- TABLE 1 GLOSSARY OF GENETIC TERMS
GENES AND FAMILY HISTORY
OVERVIEW OF GENETIC DISORDERS
GENE MUTATIONS
CAUSES OF GENE MUTATIONS
GENETIC TESTING AND SCREENING
TESTING POPULATION GROUPS (ETHNOGENETICS)
DIRECT TO CONSUMER GENETIC TESTING
U.S. DEMOGRAPHICS
- TABLE 2 NUMBER OF BIRTHS IN THE U.S., 2006-2010
- TABLE 3 POPULATION GROWTH AND BIRTH STATISTICS U.S., 2011-2020 (THOUSANDS)
- FIGURE 1 POPULATION GROWTH AND BIRTH STATISTICS U.S. 2011-2020 (THOUSANDS)
INTERNATIONAL DEMOGRAPHICS
- TABLE 4 ESTIMATED WORLD POPULATION AND ANNUAL GROWTH BY SELECTED AGE GROUP AND SELECTED YEARS, 2000 THROUGH 2030 (MILLIONS)
- FIGURE 2 ESTIMATED WORLD POPULATION BY SELECTED AGE GROUP AND SELECTED YEARS, 2000-2030 (MILLIONS)
- TABLE 5 WORLD POPULATION BY SELECTED COUNTRIES, THROUGH 2014 (MILLIONS)
- FIGURE 3 WORLD POPULATION BY SELECTED COUNTRIES, 2007-2014 (MILLIONS)
INTERNATIONAL BIRTH RATE STATISTICS
- TABLE 6 BIRTHS FOR SELECTED COUNTRIES, 1995-2015 (THOUSANDS)
- FIGURE 4 BIRTHS FOR SELECTED COUNTRIES, 1995-2015 (THOUSANDS)
CHAPTER FOUR: ISSUES AFFECTING THE MARKET
GENETIC RESEARCH
GENE THERAPY COMPARED TO CELL THERAPY
ISSUES WITH HUMAN CLONING
LEGISTLATIVE ISSUES
THE GENETIC INFORMATION NONDISCRIMINATION ACT (GINA)
GERMAN LEGISLATION
AUSTRALIAN GENETIC TESTING LAW
EUROPEAN UNION ACTION IN THE FIELD OF RARE DISEASES
GENETICS CLINICS
- TABLE 7 NUMBER OF GENETIC CLINICS BY COUNTRY
GENOME SEQUENCING TECHNOLOGY
TECHNOLOGICAL APPLICATIONS OF GENOME SEQUENCING
SEQUENCING COSTS
CHAPTER FIVE: GENE MUTATION DISORDERS
DESCRIPTIONS OF GENE MUTATION DISORDERS
CYSTIC FIBROSIS
Treatment Options
Key Products
- TABLE 8 PHARMACEUTICAL TREATMENTS FOR CYSTIC FIBROSIS
New Developments
- TABLE 9 CYSTIC FIBROSIS DEVELOPMENTS BY PHASE OF DEVELOPMENT
FABRY DISEASE
Treatment Options
Key Products
- TABLE 10 PHARMACEUTICAL TREATMENTS FOR FABRY DISEASE
New Developments
- TABLE 11 FABRY DISEASE DEVELOPMENTS BY PHASE OF DEVELOPMENT
GAUCHER’S DISEASE
Treatment Options
Key Products
- TABLE 12 PHARMACEUTICAL TREATMENTS FOR GAUCHER’S DISEASE
New Developments
- TABLE 13 GAUCHER’S DISEASE DEVELOPMENTS BY PHASE OF DEVELOPMENT
HEMOPHILIA AND OTHER INHERITED BLEEDING DISORDERS
Treatment Options
Key Products
- TABLE 14 BIOLOGICAL AND PHARMACEUTICAL TREATMENTS FOR HEMOPHILIA AND OTHERS
- TABLE 15 EXPANDED INDICATIONS AND PRODUCT CHANGES 2005-2009
New Developments
- TABLE 16 HEMOPHILIA AND RELATED BLOOD DISORDER DEVELOPMENTS BY PHASE OF DEVELOPMENT
HUNTINGTON’S DISEASE
Treatment Options
Key Products
- TABLE 17 PHARMACEUTICAL TREATMENTS FOR HUNTINGTON’S DISEASE
New Developments
- TABLE 18 HUNTINGTON’S DISEASE DEVELOPMENTS BY PHASE OF DEVELOPMENT
HUNTER SYNDROME
Treatment Options
Key Products
- TABLE 19 PHARMACEUTICAL TREATMENTS FOR HUNTER SYNDROME
New Developments
- TABLE 20 HUNTER SYNDROME DEVELOPMENTS BY PHASE OF DEVELOPMENT
HURLER SYNDROME
Treatment Options
Key Products
- TABLE 21 PHARMACEUTICAL TREATMENTS FOR HURLER SYNDROME
New Developments
MAROTEAUX-LAMY SYNDROME
Treatment Options
Key Products
- TABLE 22 PHARMACEUTICAL TREATMENTS FOR MAROTEAUX-LAMY SYNDROME
PHENYLKETONURIA (PKU)
Treatment Options
Key Products
- TABLE 23 PHARMACEUTICAL TREATMENTS FOR PKU
New Developments
- TABLE 24 PKU DEVELOPMENTS BY PHASE OF DEVELOPMENT
POMPE DISEASE
Treatment Options
Key Products
- TABLE 25 PHARMACEUTICAL TREATMENTS FOR POMPE DISEASE
New Developments
- TABLE 26 POMPE DISEASE DEVELOPMENTS BY PHASE OF DEVELOPMENT
SICKLE CELL DISEASE
Treatment Options
Key Products
- TABLE 27 PHARMACEUTICAL TREATMENTS FOR SICKLE CELL DISEASE
New Developments
- TABLE 28 SICKLE CELL DISEASE DEVELOPMENTS BY PHASE OF DEVELOPMENT
TAY-SACHS DISEASE
Treatment Options
Key Products
New Developments
- TABLE 29 TAY-SACHS DISEASE DEVELOPMENTS BY PHASE OF DEVELOPMENT
WILSON’S DISEASE
Treatment Options
Key Products
- TABLE 30 PHARMACEUTICAL TREATMENTS FOR WILSON’S DISEASE
New Developments
- TABLE 31 WILSON’S DISEASE DEVELOPMENTS BY PHASE OF DEVELOPMENT
OTHER APPROVED PRODUCTS FOR RARE GENETIC DISORDERS
OTHER NEW DEVELOPMENT PROJECTS
PHASE II
PHASE I
PRECLINICAL
MUSCULAR DYSTROPHY FOCUS: PRECLINICAL THROUGH PHASE III
AVI-4658
AVI-5038
Catena/Sovrima
PRO044
PTC-124
SNT-317/omigapil
STATISTICS
CYSTIC FIBROSIS
- TABLE 32 CYSTIC FIBROSIS STATISTICS
GAUCHER’S DISEASE
- TABLE 33 GAUCHER’S DISEASE STATISTICS
PHENYLKETONURIA
TAY-SACHS DISEASE
- TABLE 34 TAY-SACHS DISEASE STATISTICS
SICKLE CELL DISEASE
- TABLE 35 SICKLE-CELL DISEASE STATISTICS
HEMOPHILIA
HUNTINGTON’S DISEASE
MARKET SIZE AND GROWTH
- TABLE 36 GLOBAL GENE MUTATION PRODUCT REVENUES BY DISORDER THROUGH 2014 ($ MILLIONS)
- FIGURE 5 GLOBAL GENE MUTATION PRODUCT REVENUES BY DISORDER, 2007-2014 ($ MILLIONS)
COMPETITIVE STRUCTURE AND MARKET SHARE
CYSTIC FIBROSIS MARKET
- TABLE 37 GLOBAL PRODUCT REVENUES FOR CYSTIC FIBROSIS TREATMENTS BY MANUFACTURER, THROUGH 2014 ($ MILLIONS)
- FIGURE 6 GLOBAL PRODUCT REVENUES FOR CYSTIC FIBROSIS TREATMENTS BY MANUFACTURER, 2007-2014 ($ MILLIONS)
FABRY DISEASE MARKET
- TABLE 38 GLOBAL PRODUCT REVENUES FOR FABRY DISEASE TREATMENTS BY MANUFACTURER, THROUGH 2014 ($ MILLIONS)
- FIGURE 7 GLOBAL PRODUCT REVENUES FOR FABRY DISEASE TREATMENTS BY MANUFACTURER, 2007-2014 ($ MILLIONS)
GAUCHER’S DISEASE MARKET
- TABLE 39 GLOBAL PRODUCT REVENUES FOR GAUCHER’S DISEASE TREATMENTS BY MANUFACTURER, THROUGH 2014 ($ MILLIONS)
- FIGURE 8 GLOBAL PRODUCT REVENUES FOR GAUCHER’S DISEASE TREATMENTS BY MANUFACTURER, 2007-2014 ($ MILLIONS)
HEMOPHILIA AND RELATED BLOOD DISORDER MARKET
- TABLE 40 GLOBAL PRODUCT REVENUES FOR HEMOPHILIA TREATMENTS BY MANUFACTURER, THROUGH 2014 ($ MILLIONS)
- FIGURE 9 GLOBAL PRODUCT REVENUES FOR HEMOPHILIA TREATMENTS BY MANUFACTURER, 2007-2014 ($ MILLIONS)
HUNTINGTON’S DISEASE MARKET
- TABLE 41 GLOBAL PRODUCT REVENUES FOR HUNTINGTON’S DISEASE TREATMENTS BY MANUFACTURER, THROUGH 2014 ($ MILLIONS)
- FIGURE 10 GLOBAL PRODUCT REVENUES FOR HUNTINGTON’S DISEASE TREATMENTS BY MANUFACTURER, 2007-2014 ($ MILLIONS)
HUNTER SYNDROME MARKET
- TABLE 42 GLOBAL PRODUCT REVENUES FOR HUNTER SYNDROME TREATMENTS BY MANUFACTURER, THROUGH 2014 ($ MILLIONS)
- FIGURE 11 GLOBAL PRODUCT REVENUES FOR HUNTER SYNDROME TREATMENTS BY MANUFACTURER, 2007-2014 ($ MILLIONS)
HURLER SYNDROME MARKET
- TABLE 43 GLOBAL PRODUCT REVENUES FOR HURLER SYNDROME TREATMENTS BY MANUFACTURER, THROUGH 2014 ($ MILLIONS)
- FIGURE 12 GLOBAL PRODUCT REVENUES FOR HURLER SYNDROME TREATMENTS BY MANUFACTURER, 2007-2014 ($ MILLIONS)
MAROTEAUX-LAMY SYNDROME MARKET
- TABLE 44 GLOBAL PRODUCT REVENUES FOR MAROTEAUX-LAMY SYNDROME TREATMENTS BY MANUFACTURER, THROUGH 2014 ($ MILLIONS)
- FIGURE 13 GLOBAL PRODUCT REVENUES FOR MAROTEAUX-LAMY SYNDROME TREATMENTS BY MANUFACTURER, 2007-2014 ($ MILLIONS)
PHENYLKETONURIA (PKU) MARKET
- TABLE 45 GLOBAL PRODUCT REVENUES FOR PKU TREATMENTS BY MANUFACTURER, THROUGH 2014 ($ MILLIONS)
- FIGURE 14 GLOBAL PRODUCT REVENUES FOR PKU TREATMENTS BY MANUFACTURER, 2007-2014 ($ MILLIONS)
POMPE DISEASE MARKET
- TABLE 46 GLOBAL PRODUCT REVENUES FOR POMPE DISEASE TREATMENTS BY MANUFACTURER, THROUGH 2014 ($ MILLIONS)
- FIGURE 15 GLOBAL PRODUCT REVENUES FOR POMPE DISEASE TREATMENTS BY MANUFACTURER, 2007-2014 ($ MILLIONS)
CHAPTER SIX: CHROMOSONAL DISORDERS
DESCRIPTIONS OF CHROMOSONAL DISORDERS
DOWN SYNDROME
Treatment Options
New Developments
- TABLE 47 DOWN SYNDROME DEVELOPMENTS BY PHASE OF DEVELOPMENT
KLINEFELTER SYNDROME
Treatment Options
Key Products
- TABLE 48 EXAMPLES OF PHARMACEUTICAL TREATMENTS FOR KLINEFELTER SYNDROME
New Developments
- TABLE 49 EXAMPLES OF TESTOSTERONE REPLACEMENT DEVELOPMENTS BY PHASE OF DEVELOPMENT
PRADER-WILLI SYNDROME
Treatment Options
Key Products
- TABLE 50 PHARMACEUTICAL TREATMENTS FOR PRADER-WILLI SYNDROME
New Developments
TURNER SYNDROME
Treatment Options
Key Products
- TABLE 51 FDA APPROVED BIOPHARMACEUTICAL TREATMENTS FOR TURNER’S SYNDROME
New Developments
- TABLE 52 TURNER SYNDROME DEVELOPMENTS BY PHASE OF DEVELOPMENT
STATISTICS
DOWN SYNDROME
- TABLE 53 DOWN SYNDROME RISK AS A COMPARISON TO MATERNAL AGE
KLINEFELTER SYNDROME
PRADER-WILLI SYNDROME
TURNER SYNDROME
MARKET SIZE AND GROWTH
- TABLE 54 GLOBAL CHROMOSONAL DISORDER PRODUCT REVENUES* BY DISORDER, THROUGH 2014 ($ MILLIONS)
- FIGURE 16 GLOBAL CHROMOSONAL DISORDER PRODUCT REVENUES BY DISORDER, 2007-2014 ($ MILLIONS)
COMPETITIVE STRUCTURE AND MARKET SHARE
KLINEFELTER SYNDROME
- TABLE 55 GLOBAL PRODUCT REVENUES FOR KLINEFELTER SYNDROME TREATMENTS, THROUGH 2014 ($ MILLIONS)
- FIGURE 17 GLOBAL PRODUCT REVENUES FOR KLINEFELTER SYNDROME TREATMENTS BY MANUFACTURER, 2007-2014 ($ MILLIONS)
PRADER-WILLI SYNDROME
- TABLE 56 GLOBAL PRODUCT REVENUES* FOR PRADER-WILLI SYNDROME TREATMENTS BY MANUFACTURER, THROUGH 2014 ($ MILLIONS)
- FIGURE 18 GLOBAL PRODUCT REVENUES FOR PRADER-WILLI SYNDROME TREATMENTS BY MANUFACTURER, 2007-2014 ($ MILLIONS)
TURNER’S SYNDROME
- TABLE 57 GLOBAL PRODUCT REVENUES* FOR TURNER’S SYNDROME TREATMENTS BY MANUFACTURER, THROUGH 2014 ($ MILLIONS)
- FIGURE 19 GLOBAL PRODUCT REVENUES FOR TURNER’S SYNDROME TREATMENTS BY MANUFACTURER, 2007-2014 ($ MILLIONS)
CHAPTER SEVEN: MARKET SUMMARY
TOTAL GLOBAL MARKET FOR GENETIC DISORDERS
- TABLE 58 GLOBAL GENETIC DISORDER PRODUCT REVENUES BY MAJOR MARKET SEGMENT, THROUGH 2014 ($ MILLIONS)
- FIGURE 20 GLOBAL GENETIC DISORDER PRODUCT REVENUES BY MAJOR MARKET SEGMENT, 2007-2014 ($ MILLIONS)
TOTAL GLOBAL MARKET AND PERCENT DISTRIBUTION BY SELECTED GENETIC DISORDER
- TABLE 59 GLOBAL GENETIC DISORDER PRODUCT REVENUES BY DISORDER THROUGH 2014 ($ MILLIONS)
- FIGURE 21 GLOBAL GENETIC DISORDER PRODUCT REVENUES BY DISORDER, 2007-2014 ($ MILLIONS)
GEOGRAPHICAL ANALYSIS OF GENETIC DISORDER TREATMENTS
- TABLE 60 GLOBAL GENETIC DISORDER PRODUCT REVENUES BY SELECTED COUNTRY, THROUGH 2014 ($ MILLIONS)
- FIGURE 22 GLOBAL GENETIC DISORDER PRODUCT REVENUES BY SELECTED COUNTRY, 2007-2014 ($ MILLIONS)
MAJOR INDUSTRY PARTICIPANTS BY PRODUCT SEGMENT
- TABLE 61 MAJOR INDUSTRY PARTICIPANTS BY PRODUCT SEGMENTS AND SALES GROWTH, THROUGH 2014 ($ MILLIONS)
CHAPTER EIGHT: TECHNOLOGY
PATENT ANALYSIS
PATENTS AND GENETICS
PATENT EVALUATION BY GENETIC CONDITION
- TABLE 62 PATENT INFORMATION BY GENETIC CONDITION
- FIGURE 23 PERCENT OF PATENTS BY GENETIC CONDITION (%)
PATENT EVALUATION BY ASSIGNEE
- TABLE 63 GENETIC PATENT ASSIGNEES
Bristol-Myers Squibb
Eli Lilly
G. D. Searle & Co./Monsanto Company
Genzyme Corp.
Purdue Pharma
Sigma-Tau Industrie Farmaceutiche Riunite S.p.A.
Transkaryotic Therapies
Wyeth
CHAPTER NINE: COMPANY PROFILES
BAXTER INTERNATIONAL, INC.
- TABLE 64 BAXTER’S COMPANY REVENUES, 2007-2009 ($ MILLIONS)
- FIGURE 24 BAXTER’S COMPANY REVENUES, 2007-2009 ($ MILLIONS)
BAYER AG
- TABLE 65 BAYER’S COMPANY REVENUES, 2007-2009 ($ MILLIONS)
- FIGURE 25 BAYER’S COMPANY REVENUES, 2007-2009 ($ MILLIONS)
BIOMARIN PHARMACEUTICAL, INC.
- TABLE 66 BIOMARIN’S COMPANY REVENUES, 2007-2009 ($ MILLIONS)
- FIGURE 26 BIOMARIN’S COMPANY REVENUES, 2007-2009 ($ MILLIONS)
CANGENE CORP.
- TABLE 67 CANGENE’S COMPANY REVENUES, 2007-2009* ($ MILLIONS)
- FIGURE 27 CANGENE’S COMPANY REVENUES, 2007-2009 ($ MILLIONS)
CSL BEHRING (CSL LIMITED)
- TABLE 68 CSL’S COMPANY REVENUES, 2007-2009* ($ MILLIONS)
- FIGURE 28 CSL’S COMPANY REVENUES, 2007-2009 ($ MILLIONS)
ELI LILLY
- TABLE 69 ELI LILLY’S COMPANY REVENUES, 2007-2009 ($ MILLIONS)
- FIGURE 29 ELI LILLY’S COMPANY REVENUES, 2007-2009 ($ MILLIONS)
ENDO PHARMACEUTICALS
- TABLE 70 ENDO’S COMPANY REVENUES, 2007-2009 ($ MILLIONS)
- FIGURE 30 ENDO’S COMPANY REVENUES, 2007-2009 ($ MILLIONS)
GENENTECH INC.
- TABLE 71 GENENTECH AND ROCHE COMPANY REVENUES, 2007-2009 ($ MILLIONS)
- FIGURE 31 GENENTECH AND ROCHE COMPANY REVENUES, 2007-2009 ($ MILLIONS)
GENZYME
- TABLE 72 GENZYME’S COMPANY REVENUES, 2007-2009 ($ MILLIONS)
- FIGURE 32 GENZYME’S COMPANY REVENUES, 2007-2009 ($ MILLIONS)
H. LUNDBECK A/S
- TABLE 73 LUNDBECK’S COMPANY REVENUES, 2007-2009 ($ MILLIONS)
- FIGURE 33 LUNDBECK’S COMPANY REVENUES, 2007-2009 ($ MILLIONS)
NOVARTIS INTERNATIONAL AG/SANDOZ
- TABLE 74 NOVARTIS’ COMPANY REVENUES, 2007-2009 ($ MILLIONS)
- FIGURE 34 NOVARTIS’ COMPANY REVENUES, 2007-2009 ($ MILLIONS)
NOVO NORDISK A/S
- TABLE 75 NOVO NORDISK’S COMPANY REVENUES, 2007-2009 ($ MILLIONS)
- FIGURE 35 NOVO NORDISK’S COMPANY REVENUES, 2007-2009 ($ MILLIONS)
PFIZER, INC/WYETH
- TABLE 76 PFIZER’S COMPANY REVENUES, 2007-2009 ($ MILLIONS)
- FIGURE 36 PFIZER’S COMPANY REVENUES, 2007-2009 ($ MILLIONS)
SHIRE PLC
- TABLE 77 SHIRE’S COMPANY REVENUES, 2007-2009 ($ MILLIONS)
- FIGURE 37 SHIRE’S COMPANY REVENUES, 2007-2009 ($ MILLIONS)
APPENDIX I: COMPANIES WITH GENETIC RESEARCH PROJECTS
ADEONA PHARMACEUTICALS, INC.
AEROVANCE, INC.
ALDAGEN, INC.
AMICUS THERAPEUTICS, INC.
AOP ORPHAN PHARMACEUTICALS AG
APPLIED GENETIC TECHNOLOGIES CORP.
AVI BIOPHARMA
AVICENA GROUP, INC.
BAYER SCHERING PHARMA AG
BIOGEN IDEC
BIOMARIN PHARMACEUTICAL INC.
COPERNICUS THERAPEUTICS, INC
CSL BEHRING
EISAI, INC.
GILEAD SCIENCES, INC.
GLAXOSMITHKLINE PLC
HOLLIS-EDEN PHARMACEUTICALS, INC.
INSPIRE PHARMACEUTICALS, INC.
IPSEN
KALOBIOS PHARMACEUTICALS, INC.
LANTIBIO, INC.
MPEX PHARMACEUTICALS, INC.
NEUROSEARCH A/S
NORTHFIELD LABORATORIES, INC.
NOVO NORDISK A/S
PFIZER, INC.
PHARMAXIS LTD
PROSENSA HOLDING B.V.
PROTALIX BIOTHERAPEUTICS
PTC THERAPEUTICS, INC.
QUATRX PHARMACEUTICALS
REGENERX BIOPHARMACEUTICALS, INC.
REPROS THERAPEUTICS INC.
SANTHERA PHARMACEUTICALS HOLDING AG
SHIRE PLC
SUCAMPO PHARMACEUTICALS, INC.
TRANSAVE, INC.
VERTEX PHARMACEUTICALS, INC.
APPENDIX II: ASSOCIATIONS AND ORGANIZATIONS
ANGELMAN SYNDROME FOUNDATION, INC.
CANADIAN SOCIETY FOR MUCOPOLYSACCHARIDE AND RELATED DISEASES, INC
CHILDREN LIVING WITH INHERITED METABOLIC DISEASES (CLIMB)
CELIAC DISEASE FOUNDATION
CELIAC SPRUE ASSOCIATION
CURE CMD (CONGENITAL MUSCULAR DYSTROPHY)
CYSTIC FIBROSIS FOUNDATION
CYSTIC FIBROSIS RESEARCH, INC.
CYSTIC FIBROSIS TRUST
CYSTIC FIBROSIS WORLDWIDE
DOWN'S SYNDROME ASSOCIATION (UK)
FABRY SUPPORT & INFORMATION GROUP
GAUCHERS ASSOCIATION (UK)
HIDE & SEEK FOUNDATION FOR LYSOSOMAL DISEASE RESEARCH
HEMOPHILIA FEDERATION OF AMERICA
HUNTINGTON’S ASSOCIATION OF SOUTH AFRICA POSTBOX 4640
HUNTINGTON’S DISEASE SOCIETY OF AMERICA
HUNTINGTON SOCIETY OF CANADA
INTERNATIONAL CENTER FOR FABRY DISEASE
INSTITUTO DE ERRORES INNATOS DEL METABOLISMO
KLINEFELTER SYNDROME & ASSOCIATES (KS&A)
MARCH OF DIMES BIRTH DEFECTS FOUNDATION
MUSCULAR DYSTROPHY ASSOCIATION
MUSCULAR DYSTROPHY LIMITED (AUSTRALIA)
MUSCULAR DYSTROPHY ASSOCIATION OF NEW ZEALAND, INC.
MUSCULAR DYSTROPHY CAMPAIGN
MUSCULAR DYSTROPHY CANADA
MUSCULAR DYSTROPHY IRELAND
NATIONAL FOUNDATION FOR CELIAC AWARENESS (NFCA)
NATIONAL GAUCHER’S FOUNDATION
NATIONAL HEMOPHILIA FOUNDATION
NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE (NINDS)
NATIONAL MPS SOCIETY (HUNTER SYNDROME)
NATIONAL MPS (MUCOPOLYSACCHARIDOSES/MUCOLIPIDOSES) SOCIETY, INC.
NATIONAL ORGANIZATION FOR RARE DISORDERS (NORD)
NATIONAL TAY-SACHS AND ALLIED DISEASES ASSOCIATION, INC.
PRADER-WILLI SYNDROME ASSOCIATION (UK)
THE PRADER-WILLI SYNDROME ASSOCIATION (USA)
SICKLE CELL ASSOCIATION
SICKLE CELL ASSOCIATION OF ONTARIO
SICKLE CELL ASSOCIATION OF TEXAS GULF COAST
SICKLE CELL DISEASE ASSOCIATION OF PIEDMONT
SICKLE CELL DISEASE FOUNDATION OF CALIFORNIA
SOCIETY FOR MUSCULAR DYSTROPHY INFORMATION INTERNATIONAL
TURNER'S SYNDROME SOCIETY
TURNER SYNDROME SUPPORT SOCIETY (UK)
U.S. NATIONAL LIBRARY OF MEDICINE
APPENDIX III: INTERNATIONAL LOCATIONS OF GENETIC CLINICS
ARGENTINA
AUSTRALIA
AUSTRIA
BAHRAIN
BELGIUM
BRAZIL
CANADA
CHILE
CZECH REPUBLIC
DENMARK
FRANCE
GERMANY
GREECE
INDIA
ISRAEL
ITALY
JAPAN
NETHERLANDS
NEW ZEALAND
PORTUGAL
RUSSIA
SAUDI ARABIA
SOUTH KOREA
SPAIN
TURKEY
UNITED KINGDOM
LIST OF TABLES
SUMMARY TABLE A GLOBAL GENETIC DISORDER PRODUCT
REVENUES BY MAJOR MARKET SEGMENT, THROUGH 2014 ($
MILLIONS)
SUMMARY TABLE B TOTAL COMPANY SALES FOR GENETIC
DISORDER TREATMENT, 2009 ($ MILLIONS)
TABLE 1 GLOSSARY OF GENETIC TERMS
TABLE 2 NUMBER OF BIRTHS IN THE U.S., 2006-2010
TABLE 3 POPULATION GROWTH AND BIRTH STATISTICS U.S., 2011-
2020 (THOUSANDS)
TABLE 4 ESTIMATED WORLD POPULATION AND ANNUAL GROWTH
BY SELECTED AGE GROUP AND SELECTED YEARS, 2000
THROUGH 2030 (MILLIONS)
TABLE 5 WORLD POPULATION BY SELECTED COUNTRIES,
THROUGH 2014 (MILLIONS)
TABLE 6 BIRTHS FOR SELECTED COUNTRIES, 1995-2015
(THOUSANDS)
TABLE 7 NUMBER OF GENETIC CLINICS BY COUNTRY
TABLE 8 PHARMACEUTICAL TREATMENTS FOR CYSTIC FIBROSIS
TABLE 9 CYSTIC FIBROSIS DEVELOPMENTS BY PHASE OF
DEVELOPMENT
TABLE 10 PHARMACEUTICAL TREATMENTS FOR FABRY DISEASE
TABLE 11 FABRY DISEASE DEVELOPMENTS BY PHASE OF
DEVELOPMENT
TABLE 12 PHARMACEUTICAL TREATMENTS FOR GAUCHER’S
DISEASE
TABLE 13 GAUCHER’S DISEASE DEVELOPMENTS BY PHASE OF
DEVELOPMENT
TABLE 14 BIOLOGICAL AND PHARMACEUTICAL TREATMENTS FOR
HEMOPHILIA AND OTHERS
TABLE 15 EXPANDED INDICATIONS AND PRODUCT CHANGES 2005-
2009
TABLE 16 HEMOPHILIA AND RELATED BLOOD DISORDER
DEVELOPMENTS BY PHASE OF DEVELOPMENT
TABLE 17 PHARMACEUTICAL TREATMENTS FOR HUNTINGTON’S
DISEASE
TABLE 18 HUNTINGTON’S DISEASE DEVELOPMENTS BY PHASE OF
DEVELOPMENT
TABLE 19 PHARMACEUTICAL TREATMENTS FOR HUNTER
SYNDROME
TABLE 20 HUNTER SYNDROME DEVELOPMENTS BY PHASE OF
DEVELOPMENT
TABLE 21 PHARMACEUTICAL TREATMENTS FOR HURLER
SYNDROME
TABLE 22 PHARMACEUTICAL TREATMENTS FOR MAROTEAUX-
LAMY SYNDROME
TABLE 23 PHARMACEUTICAL TREATMENTS FOR PKU
TABLE 24 PKU DEVELOPMENTS BY PHASE OF DEVELOPMENT
TABLE 25 PHARMACEUTICAL TREATMENTS FOR POMPE DISEASE
TABLE 26 POMPE DISEASE DEVELOPMENTS BY PHASE OF
DEVELOPMENT
TABLE 27 PHARMACEUTICAL TREATMENTS FOR SICKLE CELL
DISEASE
TABLE 28 SICKLE CELL DISEASE DEVELOPMENTS BY PHASE OF
DEVELOPMENT
TABLE 29 TAY-SACHS DISEASE DEVELOPMENTS BY PHASE OF
DEVELOPMENT
TABLE 30 PHARMACEUTICAL TREATMENTS FOR WILSON’S
DISEASE
TABLE 31 WILSON’S DISEASE DEVELOPMENTS BY PHASE OF
DEVELOPMENT
TABLE 32 CYSTIC FIBROSIS STATISTICS
TABLE 33 GAUCHER’S DISEASE STATISTICS
TABLE 34 TAY-SACHS DISEASE STATISTICS
TABLE 35 SICKLE-CELL DISEASE STATISTICS
TABLE 36 GLOBAL GENE MUTATION PRODUCT REVENUES BY
DISORDER THROUGH 2014 ($ MILLIONS)
TABLE 37 GLOBAL PRODUCT REVENUES FOR CYSTIC FIBROSIS
TREATMENTS BY MANUFACTURER, THROUGH 2014 ($ MILLIONS)
TABLE 38 GLOBAL PRODUCT REVENUES FOR FABRY DISEASE
TREATMENTS BY MANUFACTURER, THROUGH 2014 ($ MILLIONS)
TABLE 39 GLOBAL PRODUCT REVENUES FOR GAUCHER’S DISEASE
TREATMENTS BY MANUFACTURER, THROUGH 2014 ($ MILLIONS)
TABLE 40 GLOBAL PRODUCT REVENUES FOR HEMOPHILIA
TREATMENTS BY MANUFACTURER, THROUGH 2014 ($ MILLIONS)
TABLE 41 GLOBAL PRODUCT REVENUES FOR HUNTINGTON’S
DISEASE TREATMENTS BY MANUFACTURER, THROUGH 2014 ($
MILLIONS)
TABLE 42 GLOBAL PRODUCT REVENUES FOR HUNTER SYNDROME
TREATMENTS BY MANUFACTURER, THROUGH 2014 ($ MILLIONS)
TABLE 43 GLOBAL PRODUCT REVENUES FOR HURLER SYNDROME
TREATMENTS BY MANUFACTURER, THROUGH 2014 ($ MILLIONS)
TABLE 44 GLOBAL PRODUCT REVENUES FOR MAROTEAUX-LAMY
SYNDROME TREATMENTS BY MANUFACTURER, THROUGH 2014 ($
MILLIONS)
TABLE 45 GLOBAL PRODUCT REVENUES FOR PKU TREATMENTS BY
MANUFACTURER, THROUGH 2014 ($ MILLIONS)
TABLE 46 GLOBAL PRODUCT REVENUES FOR POMPE DISEASE
TREATMENTS BY MANUFACTURER, THROUGH 2014 ($ MILLIONS)
TABLE 47 DOWN SYNDROME DEVELOPMENTS BY PHASE OF
DEVELOPMENT
TABLE 48 EXAMPLES OF PHARMACEUTICAL TREATMENTS FOR
KLINEFELTER SYNDROME
TABLE 49 EXAMPLES OF TESTOSTERONE REPLACEMENT
DEVELOPMENTS BY PHASE OF DEVELOPMENT
TABLE 50 PHARMACEUTICAL TREATMENTS FOR PRADER-WILLI
SYNDROME
TABLE 51 FDA APPROVED BIOPHARMACEUTICAL TREATMENTS
FOR TURNER’S SYNDROME
TABLE 52 TURNER SYNDROME DEVELOPMENTS BY PHASE OF
DEVELOPMENT
TABLE 53 DOWN SYNDROME RISK AS A COMPARISON TO
MATERNAL AGE
TABLE 54 GLOBAL CHROMOSONAL DISORDER PRODUCT
REVENUES* BY DISORDER, THROUGH 2014 ($ MILLIONS)
TABLE 55 GLOBAL PRODUCT REVENUES FOR KLINEFELTER
SYNDROME TREATMENTS, THROUGH 2014 ($ MILLIONS)
TABLE 56 GLOBAL PRODUCT REVENUES* FOR PRADER-WILLI
SYNDROME TREATMENTS BY MANUFACTURER, THROUGH 2014 ($
MILLIONS)
TABLE 57 GLOBAL PRODUCT REVENUES* FOR TURNER’S
SYNDROME TREATMENTS BY MANUFACTURER, THROUGH 2014 ($
MILLIONS)
TABLE 58 GLOBAL GENETIC DISORDER PRODUCT REVENUES BY
MAJOR MARKET SEGMENT, THROUGH 2014 ($ MILLIONS)
TABLE 59 GLOBAL GENETIC DISORDER PRODUCT REVENUES BY
DISORDER THROUGH 2014 ($ MILLIONS)
TABLE 60 GLOBAL GENETIC DISORDER PRODUCT REVENUES BY
SELECTED COUNTRY, THROUGH 2014 ($ MILLIONS)
TABLE 61 MAJOR INDUSTRY PARTICIPANTS BY PRODUCT
SEGMENTS AND SALES GROWTH, THROUGH 2014 ($ MILLIONS)
TABLE 62 PATENT INFORMATION BY GENETIC CONDITION
TABLE 63 GENETIC PATENT ASSIGNEES
TABLE 64 BAXTER’S COMPANY REVENUES, 2007-2009 ($ MILLIONS)
TABLE 65 BAYER’S COMPANY REVENUES, 2007-2009 ($ MILLIONS)
TABLE 66 BIOMARIN’S COMPANY REVENUES, 2007-2009 ($
MILLIONS)
TABLE 67 CANGENE’S COMPANY REVENUES, 2007-2009* ($
MILLIONS)
TABLE 68 CSL’S COMPANY REVENUES, 2007-2009* ($ MILLIONS)
TABLE 69 ELI LILLY’S COMPANY REVENUES, 2007-2009 ($ MILLIONS)
TABLE 70 ENDO’S COMPANY REVENUES, 2007-2009 ($ MILLIONS)
TABLE 71 GENENTECH AND ROCHE COMPANY REVENUES, 2007-
2009 ($ MILLIONS)
TABLE 72 GENZYME’S COMPANY REVENUES, 2007-2009 ($ MILLIONS)
TABLE 73 LUNDBECK’S COMPANY REVENUES, 2007-2009 ($
MILLIONS)
TABLE 74 NOVARTIS’ COMPANY REVENUES, 2007-2009 ($ MILLIONS)
TABLE 75 NOVO NORDISK’S COMPANY REVENUES, 2007-2009 ($
MILLIONS)
TABLE 76 PFIZER’S COMPANY REVENUES, 2007-2009 ($ MILLIONS)
TABLE 77 SHIRE’S COMPANY REVENUES, 2007-2009 ($ MILLIONS)
LIST OF FIGURES
SUMMARY FIGURE GLOBAL GENETIC DISORDER PRODUCT
REVENUES, 2007-2014 ($ MILLIONS)
FIGURE 1 POPULATION GROWTH AND BIRTH STATISTICS U.S. 2011-
2020 (THOUSANDS)
FIGURE 2 ESTIMATED WORLD POPULATION BY SELECTED AGE
GROUP AND SELECTED YEARS, 2000-2030 (MILLIONS)
FIGURE 3 WORLD POPULATION BY SELECTED COUNTRIES, 2007-
2014 (MILLIONS)
FIGURE 4 BIRTHS FOR SELECTED COUNTRIES, 1995-2015
(THOUSANDS)
FIGURE 5 GLOBAL GENE MUTATION PRODUCT REVENUES BY
DISORDER, 2007-2014 ($ MILLIONS)
FIGURE 6 GLOBAL PRODUCT REVENUES FOR CYSTIC FIBROSIS
TREATMENTS BY MANUFACTURER, 2007-2014 ($ MILLIONS)
FIGURE 7 GLOBAL PRODUCT REVENUES FOR FABRY DISEASE
TREATMENTS BY MANUFACTURER, 2007-2014 ($ MILLIONS)
FIGURE 8 GLOBAL PRODUCT REVENUES FOR GAUCHER’S DISEASE
TREATMENTS BY MANUFACTURER, 2007-2014 ($ MILLIONS)
FIGURE 9 GLOBAL PRODUCT REVENUES FOR HEMOPHILIA
TREATMENTS BY MANUFACTURER, 2007-2014 ($ MILLIONS)
FIGURE 10 GLOBAL PRODUCT REVENUES FOR HUNTINGTON’S
DISEASE TREATMENTS BY MANUFACTURER, 2007-2014 ($
MILLIONS)
FIGURE 11 GLOBAL PRODUCT REVENUES FOR HUNTER SYNDROME
TREATMENTS BY MANUFACTURER, 2007-2014 ($ MILLIONS)
FIGURE 12 GLOBAL PRODUCT REVENUES FOR HURLER SYNDROME
TREATMENTS BY MANUFACTURER, 2007-2014 ($ MILLIONS)
FIGURE 13 GLOBAL PRODUCT REVENUES FOR MAROTEAUX-LAMY
SYNDROME TREATMENTS BY MANUFACTURER, 2007-2014 ($
MILLIONS)
FIGURE 14 GLOBAL PRODUCT REVENUES FOR PKU TREATMENTS
BY MANUFACTURER, 2007-2014 ($ MILLIONS)
FIGURE 15 GLOBAL PRODUCT REVENUES FOR POMPE DISEASE
TREATMENTS BY MANUFACTURER, 2007-2014 ($ MILLIONS)
FIGURE 16 GLOBAL CHROMOSONAL DISORDER PRODUCT
REVENUES BY DISORDER, 2007-2014 ($ MILLIONS)
FIGURE 17 GLOBAL PRODUCT REVENUES FOR KLINEFELTER
SYNDROME TREATMENTS BY MANUFACTURER, 2007-2014 ($
MILLIONS)
FIGURE 18 GLOBAL PRODUCT REVENUES FOR PRADER-WILLI
SYNDROME TREATMENTS BY MANUFACTURER, 2007-2014 ($
MILLIONS)
FIGURE 19 GLOBAL PRODUCT REVENUES FOR TURNER’S
SYNDROME TREATMENTS BY MANUFACTURER, 2007-2014 ($
MILLIONS)
FIGURE 20 GLOBAL GENETIC DISORDER PRODUCT REVENUES BY
MAJOR MARKET SEGMENT, 2007-2014 ($ MILLIONS)
FIGURE 21 GLOBAL GENETIC DISORDER PRODUCT REVENUES BY
DISORDER, 2007-2014 ($ MILLIONS)
FIGURE 22 GLOBAL GENETIC DISORDER PRODUCT REVENUES BY
SELECTED COUNTRY, 2007-2014 ($ MILLIONS)
FIGURE 23 PERCENT OF PATENTS BY GENETIC CONDITION (%)
FIGURE 24 BAXTER’S COMPANY REVENUES, 2007-2009 ($ MILLIONS)
FIGURE 25 BAYER’S COMPANY REVENUES, 2007-2009 ($
MILLIONS)
FIGURE 26 BIOMARIN’S COMPANY REVENUES, 2007-2009 ($
MILLIONS)
FIGURE 27 CANGENE’S COMPANY REVENUES, 2007-2009 ($
MILLIONS)
FIGURE 28 CSL’S COMPANY REVENUES, 2007-2009 ($ MILLIONS)
FIGURE 29 ELI LILLY’S COMPANY REVENUES, 2007-2009 ($
MILLIONS)
FIGURE 30 ENDO’S COMPANY REVENUES, 2007-2009 ($ MILLIONS)
FIGURE 31 GENENTECH AND ROCHE COMPANY REVENUES, 2007-
2009 ($ MILLIONS)
FIGURE 32 GENZYME’S COMPANY REVENUES, 2007-2009 ($
MILLIONS)
FIGURE 33 LUNDBECK’S COMPANY REVENUES, 2007-2009 ($
MILLIONS)
FIGURE 34 NOVARTIS’ COMPANY REVENUES, 2007-2009 ($ MILLIONS)
FIGURE 35 NOVO NORDISK’S COMPANY REVENUES, 2007-2009 ($
MILLIONS)
FIGURE 36 PFIZER’S COMPANY REVENUES, 2007-2009 ($ MILLIONS)
FIGURE 37 SHIRE’S COMPANY REVENUES, 2007-2009 ($ MILLIONS)