$1,318.00
This Market Spotlight report covers the Duchenne muscular dystrophy market, comprising key pipeline and marketed drugs, clinical trials, upcoming and regulatory events, patent information, a 10-year disease prevalence forecast, and licensing and acquisition deals, as well as presenting drug-specific revenue forecasts.
This Market Spotlight report covers the Duchenne Muscular Dystrophy market, comprising key marketed and pipeline drugs, clinical trials, upcoming and regulatory events, patent information, a 10-year disease prevalence forecast, probability of success, recent events and analyst opinion, and licensing and acquisition deals, as well as presenting drug-specific revenue forecasts.
Key Takeaways
CONTENTS
6 OVERVIEW
7 KEY TAKEAWAYS
8 DISEASE BACKGROUND
9 TREATMENT
9 Corticosteroids
9 Genetic therapies
9 Vitamin D supplement
10 EPIDEMIOLOGY
12 MARKETED DRUGS
14 PIPELINE DRUGS
20 RECENT EVENTS AND ANALYST OPINION
20 Edasalonexent for DMD (October 26, 2020)
21 Catena for DMD (October 6, 2020)
23 SGT-001 for DMD (December 18, 2019)
24 SGT-001 for DMD (November 12, 2019)
26 Vyondys 53 for DMD (August 19, 2019)
27 CAP-1002 for DMD (July 15, 2019)
29 PF-06939926 for DMD (June 28, 2019)
32 SingleCut CRISPR-Cas9 Program (Exonics) for DMD (June 6, 2019)
34 KEY UPCOMING EVENTS
35 KEY REGULATORY EVENTS
35 Viltepso Is Second Exon 53-Skipping Therapy Approved For DMD
35 Improved EU Patient Access & Reimbursement On The Cards For Translarna
35 Another Setback For Santhera’s Idebenone DMD Hopes
35 For Sarepta, Another Exon-Skipping Drug
36 Surprise As FDA Approves Sarepta’s Duchenne Drug Vyondys 53
36 Adverse Event Concerns With Solid Biosciences’ Candidate DMD Gene Therapy
37 PROBABILITY OF SUCCESS
38 LICENSING AND ASSET ACQUISITION DEALS
38 Ultragenyx Enters Duchenne Gene Therapy Space In $295m Deal With Solid Biosciences
38 Santhera Doubles Its DMD Portfolio By Taking Option On Vamorolone
38 Sarepta Licenses Hansa Candidate As DMD/LGMD Pre-Treatment
38 Sarepta Partners With Selecta And Codiak On Gene Therapies
39 Sarepta Secures $1.15bn From Roche In Ex-US DMD Gene Therapy Deal
39 Astellas To Pay $3bn For Gene Therapy Company Audentes
40 REVENUE OPPORTUNITY
41 CLINICAL TRIAL LANDSCAPE
42 Sponsors by status
43 Sponsors by phase
44 Recent events
47 BIBLIOGRAPHY
47 Prescription information
48 APPENDIX
LIST OF FIGURES
11 Figure 1: Trends in prevalent cases of DMD, 2019–28
14 Figure 2: Overview of pipeline drugs for DMD in the US
14 Figure 3: Pipeline drugs for DMD, by company
15 Figure 4: Pipeline drugs for DMD, by drug type
15 Figure 5: Pipeline drugs for DMD, by classification
21 Figure 6: Edasalonexent for DMD (October 26, 2020): Phase III – PolarisDMD (Ages 4-7)
23 Figure 7: Catena for DMD (October 6, 2020): Phase III – SIDEROS (w/Glucocorticoid Steroids)
29 Figure 8: CAP-1002 for DMD (July 15, 2019): Phase II – HOPE-2
34 Figure 9: Key upcoming events in DMD
37 Figure 10: Probability of success in the DMD pipeline
41 Figure 11: Clinical trials in DMD
41 Figure 12: Top 10 drugs for clinical trials in DMD
42 Figure 13: Top 10 companies for clinical trials in DMD
42 Figure 14: Trial locations in DMD
43 Figure 15: DMD trials status
44 Figure 16: DMD trials sponsors, by phase
LIST OF TABLES
10 Table 1: Prevalent cases of DMD, 2019–28
13 Table 2: Marketed drugs for DMD
16 Table 3: Pipeline drugs for DMD in the US
20 Table 4: Edasalonexent for DMD (October 26, 2020)
22 Table 5: Catena for DMD (October 6, 2020)
23 Table 6: SGT-001 for DMD (December 18, 2019)
25 Table 7: SGT-001 for DMD (November 12, 2019)
26 Table 8: Vyondys 53 for DMD (August 19, 2019)
27 Table 9: CAP-1002 for DMD (July 15, 2019)
30 Table 10: PF-06939926 for DMD (June 28, 2019)
32 Table 11: SingleCut CRISPR-Cas9 Program (Exonics) for DMD (June 6, 2019)
40 Table 12: Historical global sales, by drug ($m), 2015–19
40 Table 13: Forecasted global sales, by drug ($m), 2020–24
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