Market Spotlight: Duchenne Muscular Dystrophy (DMD)
Market Spotlight: Duchenne Muscular Dystrophy (DMD)
$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.
Author: Informa Pharma Intelligence
Publisher: Datamonitor Healthcare
- Description
- Contents
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
- Datamonitor Healthcare estimates that in 2019, there were 185,900 prevalent cases of Duchenne muscular dystrophy (DMD) in males worldwide, and forecasts that number to increase to 202,400 prevalent cases by 2028.
- Approved drugs in the DMD space target glucocorticoid receptor, dystrophin gene, and RNA translation. These drugs are administered via the oral and intravenous routes.
- The majority of industry-sponsored drugs in active clinical development for DMD are in Phase II, with only one drug in the NDA/BLA phase.
- Therapies in development for DMD focus on a wide variety of targets. The majority of pipeline drugs are administered via the oral route, with the remainder being intravenous, intraarterial, subcutaneous, and inhaled formulations.
- High-impact upcoming events for drugs in the DMD space comprise topline Phase I/II trial results for SRP-5051; topline Phase II trial results for RG6356 and Translarna; topline Phase IIb trial results for VBP15; topline Phase III trial results for Exondys 51, PF06939926, Givinostat, and Vyondys 53; and an estimated PDUFA date for AMONDYS 45.
- The overall likelihood of approval of a Phase I DMD asset is 20.3%, and the average probability a drug advances from Phase III is 54.5%. Drugs, on average, take 9.1 years from Phase I to approval, compared to 9.0 years in the overall metabolic space.
- The distribution of clinical trials across Phase I–IV indicates that the majority of trials for DMD have been in the early and midphases of development, with 71% of trials in Phase I–II, and only 29% in Phase III–IV.
- The US has a substantial lead in the number of DMD clinical trials globally. The UK leads the major European markets, while Israel has the top spot in Asia.
- Sarepta has the highest number of ongoing clinical trials for DMD, with 11 trials.
- PTC Therapeutics and Sarepta lead the industry sponsors with the highest overall number of clinical trials for DMD
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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