The global Pompe market is a monopolistic market led by Sanofi Genzyme with its two approved therapies, Myozyme and Lumizyme. Considering the rare nature of the disease, the pipeline also remains low and hence, Sanofi Genzyme is expected to continue with its market leadership position over the forecast period.The market has limited treatment options, which stand as a major growth hindrance. The impact gets magnified with the high costs associated with these available therapies. These factors work negatively toward patient adherence in an already small patient pool. However, the initiatives such as patient assistance program and favorable reimbursement policies by healthcare partners should be able to partially offset the combined impact of factors discussed above and help market overcome the existing challenges over next couple of years.

PART 01: Executive summary 6

Highlights 6

PART 02: Scope of the report 7

Market overview 7

Top-vendor offerings 8

PART 03: Market research methodology 9

Research methodology 9

Economic indicators 9

PART 04: Introduction 10

Key market highlights 10

PART 05: Disease overview 12

Understanding the disease 12

Symptoms 13

Diagnosis 13

Etiology and pathogenesis 18

Biochemical pathway 18

Epidemiology 19

Management 19

Economic burden 20

PART 06: Market landscape 21

Market overview 21

Five forces analysis 26

PART 07: Market segmentation by type of Pompe disease

27

PART 08: Geographical segmentation 28

Pompe disease treatment market in EMEA 29

Pompe disease treatment market in Americas 32

Pompe disease treatment market in US 35

Pompe disease treatment market in APAC 36

PART 09: Market drivers 40

Special regulatory drug designations for orphan drugs 40

Reimbursement policies for treatment 40

Designated under ICD-10 code 41

Shorter development timelines 41

PART 10: Impact of drivers 43

PART 11: Market challenges 44

Limited patient population 44

Weak pipeline 44

Lack of long-term commitment 46

PART 12: Impact of drivers and challenges 47

PART 13: Market trends 48

Focus on gene therapy 48

Emergence of immunotherapy for Pompe disease 49

Growing public awareness 49

PART 14: Vendor landscape 50

Competitive scenario 50

Other prominent vendors 53

PART 15: Key vendor analysis 54

Amicus Therapeutics 54

Audentes Therapeutics 56

Sanofi Genzyme 58

PART 16: Appendix 62

List of abbreviations 62

PART 17: Explore Technavio 64