Market Spotlight: Duchenne Muscular Dystrophy (DMD)
Market Spotlight: Duchenne Muscular Dystrophy (DMD)
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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. Therapies in development for DMD focus on a wide variety of targets. The largest proportion 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 CAP-1002, an anticipated final data readout from the Phase IIb VISION-DMD study for VBP15, and topline Phase III trial results for fordadistrogene movaparvovec, givinostat, pamrevlumab, and Vyondys 53.
- The overall likelihood of approval of a Phase I DMD asset is 21.2%, and the average probability a drug advances from Phase III is 54.5%. Drugs, on average, take 9.2 years from Phase I to approval, compared to 9.6 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 69% of trials in Phase I–II, and only 31% 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 10 trials.
- Sarepta leads the industry sponsors with the highest overall number of clinical trials for DMD, followed by PTC Therapeutics.
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 VBP15 for DMD (June 1, 2021)
22 SRP-9001 for DMD (May 18, 2021)
24 SGT-001 for DMD (March 15, 2021)
26 RG6356 for DMD (January 7, 2021)
27 Edasalonexent for DMD (October 26, 2020)
29 Catena for DMD (October 6, 2020)
30 SGT-001 for DMD (December 18, 2019)
33 KEY UPCOMING EVENTS
34 KEY REGULATORY EVENTS
34 Sarepta’s Next-Generation Drugs Draw Attention Amid ‘Incremental’ Approval
34 Viltepso Is Second Exon 53-Skipping Therapy Approved For DMD
34 Improved EU Patient Access & Reimbursement On The Cards For Translarna
34 Another Setback For Santhera’s Idebenone DMD Hopes
36 PROBABILITY OF SUCCESS
37 LICENSING AND ASSET ACQUISITION DEALS
37 Sarepta And Genevant Collaborate For LNP Gene Editing Candidates
37 Lilly Teams With Precision On DMD, Genetic Diseases
37 Ultragenyx Enters Duchenne Gene Therapy Space In $295m Deal With Solid Biosciences
37 Santhera Doubles Its DMD Portfolio By Taking Option On Vamorolone
38 Sarepta Licenses Hansa Candidate As DMD/LGMD Pre-Treatment
38 Sarepta Partners With Selecta And Codiak On Gene Therapies
39 REVENUE OPPORTUNITY
40 CLINICAL TRIAL LANDSCAPE
41 Sponsors by status
42 Sponsors by phase
43 Recent events
48 BIBLIOGRAPHY
49 APPENDIX
LIST OF FIGURES
11 Figure 1: Trends in prevalent cases of DMD, 2019–28
14 Figure 2: Overview of pipeline drugs for DMD in the US
14 Figure 3: Pipeline drugs for DMD, by company
15 Figure 4: Pipeline drugs for DMD, by drug type
15 Figure 5: Pipeline drugs for DMD, by classification
22 Figure 6: VBP15 for DMD (June 1, 2021): Phase IIb – VISION-DMD
24 Figure 7: SRP-9001 for DMD (May 18, 2021): Phase Ib – ENDEAVOR
27 Figure 8: RG6356 for DMD (January 7, 2021): Phase II – Study 102
29 Figure 9: Edasalonexent for DMD (October 26, 2020): Phase III – PolarisDMD (Ages 4-7)
30 Figure 10: Catena for DMD (October 6, 2020): Phase III – SIDEROS (w/Glucocorticoid Steroids)
33 Figure 11: Key upcoming events in DMD
36 Figure 12: Probability of success in the DMD pipeline
40 Figure 13: Clinical trials in DMD
40 Figure 14: Top 10 drugs for clinical trials in DMD
41 Figure 15: Top 10 companies for clinical trials in DMD
41 Figure 16: Trial locations in DMD
42 Figure 17: DMD trials status
43 Figure 18: DMD trials sponsors, by phase
LIST OF TABLES
10 Table 1: Prevalent cases of DMD, 2019–28
13 Table 2: Marketed drugs for DMD
16 Table 3: Pipeline drugs for DMD in the US
20 Table 4: VBP15 for DMD (June 1, 2021)
23 Table 5: SRP-9001 for DMD (May 18, 2021)
25 Table 6: SGT-001 for DMD (March 15, 2021)
26 Table 7: RG6356 for DMD (January 7, 2021)
28 Table 8: Edasalonexent for DMD (October 26, 2020)
29 Table 9: Catena for DMD (October 6, 2020)
31 Table 10: SGT-001 for DMD (December 18, 2019)
39 Table 11: Historical global sales, by drug ($m), 2016–20
39 Table 12: Forecasted global sales, by drug ($m), 2021–25
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