Rare and Niche Diseases Catalyst Monitor - Key Event Analysis H1 2012

GlobalData’s new report, ""Rare and Niche Diseases Catalyst Monitor - Key Event Analysis H1 2012"" takes a look at rare and niche diseases, which are an area of intense interest in the global biopharmaceutical industry today. The passage of the Orphan Drug Act in the United States in 1983 was the initial impetus behind the development of products for these diseases. Since that time, similar legislation has been enacted in the UK, Europe, Japan, Singapore, and Australia. As a result of this global legislation, numerous products have been brought to market to treat diseases for which there were no existing therapies, saving countless lives worldwide. As biotechnology and pharmaceutical companies look for ways to reinvigorate their product pipelines, they are increasingly focusing on developing products for rare and niche diseases. The market for many of these diseases is virtually wide open and will continue to be a major driving force in the industry.

Scope

• AVI BioPharma’s eteplirsen, for Duchenne muscular dystrophy: expected release of unblinded data from a Phase IIb trial.
• Elelyso (taliglucerase alfa) for Gaucher disease, developed by Protalix in partnership with Pfizer: FDA decision on New Drug Application (NDA) resulted in approval.
• Talon Therapeutics’ Marqibo (Optisome-encapsulated formulation of vincristine), for adult Philadelphia chromosome negative (Ph-) acute lymphoblastic leukemia in second or greater relapse or that has progressed following two or more lines of anti-leukemia therapy: expected FDA decision on NDA.
• Amicus Therapeutics is developing Amigal (migalastat HCl) for Fabry disease in partnership with GlaxoSmithKline: expected completion of Phase II study (Study 013) in H1.
• Intermune’s Esbriet (pirfenidone), for idiopathic pulmonary fibrosis (IPF): additional launches in France, Spain, and Italy in H1.

Reasons to buy

• Understand the key therapeutic areas that are driving the rare and niche disease markets, which will enable you to strategically focus your product development.
• Develop business strategies by understanding the trends shaping and driving the global rare and niche diseases market.
• Drive revenues by understanding the key trends, innovative products and technologies, market segments and companies likely to impact the global rare and niche diseases market in the future.
• Formulate effective sales and marketing strategies by understanding the competitive landscape and by analyzing the performance of various competitors.
• Identify emerging players with potentially strong product portfolios and create effective counter-strategies to maintain or gain a competitive advantage.

Table of Contents

1 Table of Contents 2
1.1 List of Tables 3
1.2 List of Figures 3

2 AVI BioPharma’s Eteplirsen for Duchenne Muscular Dystrophy 4
2.1 Eteplirsen Overview 4
2.1.1 History and Development of Eteplirsen 4
2.2 Overview of DMD and Its Treatment 4
2.3 Catalyst Event: Release of Unblinded Phase IIb Trial Data for Eteplirsen 5
2.3.1 The Phase IIb Trial of Eteplirsen 5
2.4 Likely Outcome of the Catalyst Event 6
2.5 Phase III Study of Eteplirsen Planned 6
2.6 Anticipated Market Penetration of Eteplirsen 6
2.7 Additional Late-Stage Products for DMD 7

3 Protalix’s Elelyso (taliglucerase alfa) for Gaucher Disease 8
3.1 Elelyso Overview 8
3.1.1 History and Development of Elelyso 8
3.2 Overview of Gaucher Disease and Its Treatment 8
3.3 History of the FDA Review of Elelyso 9
3.4 Catalyst Event: Delayed PDUFA Finally Approved on May 1 10
3.4.1 Results of the Pivotal Phase III Trial of Elelyso 10
3.4.2 Follow-On Extension Study of Elelyso 10
3.4.3 Switchover Study of Elelyso 11
3.5 Likely Outcome of the Catalyst Event 11
3.6 Anticipated Market Penetration of Elelyso 11

4 Talon Therapeutics’ Marqibo for Adult Ph- ALL 13
4.1 Marqibo Overview 13
4.2 Overview of ALL and Its Treatment 13
4.3 Catalyst Event: May 13th PDUFA Date 14
4.3.1 Phase II Study of Marqibo in Ph- ALL 14
4.3.2 Additional Studies of Marqibo 14
4.4 Likely Outcome of the Catalyst Event 15
4.5 Anticipated Market Penetration of Marqibo 15

5 Amicus Therapeutics’ Amigal for Fabry Disease 16
5.1 Amigal Overview 16
5.2 Overview of Fabry Disease and Its Treatment 16
5.3 Catalyst Event: Completion of Study 013 (Fabry Disease) in H1 17
5.4 Likely Outcome of the Catalyst Event 17
5.5 Anticipated Market Penetration of Amigal 18

6 InterMune’s Esbriet for Idiopathic Pulmonary Fibrosis 19
6.1 Esbriet Overview 19
6.2 Overview of IPF and Its Treatment 19
6.3 Catalyst Event: Additional Launches of Esbriet in France, Spain, and Italy in H1 20
6.4 Likely Outcome of the Catalyst Event 20

7 Appendix 21
7.1 Bibliography 21
7.2 Abbreviations 22
7.3 Methodology 23
7.3.1 Coverage 23
7.3.2 Secondary Research 24
7.3.3 Forecasting 24
7.3.4 Expert Panel Validation 26
7.4 About GlobalData 27
7.5 Contact Us 27
7.6 Disclaimer 27

List of Tables

Table 1:    Key Exon-Skipping Drugs Being Developed by AVI BioPharma for DMD 6
Table 2:   Additional Late-Stage Products for DMD 7
Table 3:   Types of Gaucher Disease and Their Associated Characteristics 9

List of Figures

Figure 1: GlobalData Market Forecasting Model 26