CONTENTS

LIST OF FIGURES
5 EXECUTIVE SUMMARY
5 Scope of Datamonitor’s coverage of the orphan drug sector
7 Key findings
8 Definition of an orphan drug
8 Orphan drug market analysis, 2011–17
9 Orphan drug key companies
11 MARKET ANALYSIS
11 Key findings
11 Introduction
12 Orphan drug market size, 2005–17
32 COMPANY ANALYSIS
32 Key findings
33 Introduction
34 Novartis
36 Johnson & Johnson
38 Sanofi
40 Pfizer
42 AstraZeneca
43 Celgene
45 Allergan
46 Shire
48 UCB
49 Alexion
49 BioMarin
51 NPS Pharmaceuticals
52 Vertex
54 APPENDIX
54 References
55 Exchange rates
8 Figure 1: Orphan drug sales, by peer set ($m), 2011–17
10 Figure 2: Net orphan drug sales difference, by company ($m), 2011–17
12 Figure 3: Orphan drug market size ($m), 2005–17
21 Figure 4: Orphan drug sales, by therapy area ($m), 2011–17
24 Figure 5: Orphan drug sales, by molecule type ($m), 2011–17
25 Figure 6: Orphan drug sales, by peer set ($m), 2011–17
27 Figure 7: Net orphan drug sales difference, by company ($m), 2011–17

LIST OF TABLES

5 Table 1: Datamonitor’s orphan drug market company coverage
15 Table 2: Top 20 brands with an orphan drug approval by the FDA
18 Table 3: Orphan drugs therapy area sales ($m), 2011–17
23 Table 4: Orphan drug sales, by molecule type ($m), 2011–17
29 Table 5: Key orphan disease players as acquisition targets for major pharma companies
33 Table 6: The leading pharmaceutical companies in the orphan drug arena
55 Table 7: Exchange rates, 2012