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 (DMD) market, comprising key marketed and pipeline drugs, clinical trials, upcoming events, 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.
Author: Informa Pharma Intelligence
Publisher: Datamonitor Healthcare
- Description
- Contents
This Market Spotlight report covers the Duchenne Muscular Dystrophy (DMD) market, comprising key marketed and pipeline drugs, clinical trials, upcoming events, 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 majority of pipeline drugs for DMD are administered via the oral or intravenous routes, with the remainder being 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, 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 nine 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 Fordadistrogene Movaparvovec for DMD (December 20, 2021)
21 VBP15 for DMD (November 23, 2021)
24 Fordadistrogene Movaparvovec for DMD (September 28, 2021)
25 VBP15 for DMD (June 1, 2021)
28 SRP-9001 for DMD (May 18, 2021)
30 SGT-001 for DMD (March 15, 2021)
31 SRP-9001 for DMD (January 7, 2021)
33 Edasalonexent for DMD (October 26, 2020)
35 Catena for DMD (October 6, 2020)
37 KEY UPCOMING EVENTS
38 PROBABILITY OF SUCCESS
39 LICENSING AND ASSET ACQUISITION DEALS
39 Nippon Shinyaku To Commercialize Capricor’s DMD Drug In US
39 Santhera Licenses DMD Drug To Sperogenix In China
39 CANbridge Licenses Scriptr’s Gene Therapy Tech For Dystrophinopathies
39 Lilly To Try ProQR’s RNA-Editing Technology In Liver, Neurology Indications
41 REVENUE OPPORTUNITY
43 CLINICAL TRIAL LANDSCAPE
44 Sponsors by status
45 Sponsors by phase
46 Recent events
51 BIBLIOGRAPHY
52 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
24 Figure 6: VBP15 for DMD (November 23, 2021): Phase IIb – VISION-DMD
28 Figure 7: VBP15 for DMD (June 1, 2021): Phase IIb – VISION-DMD
30 Figure 8: SRP-9001 for DMD (May 18, 2021): Phase Ib – ENDEAVOR
33 Figure 9: SRP-9001 for DMD (January 7, 2021): Phase II – Study 102
35 Figure 10: Edasalonexent for DMD (October 26, 2020): Phase III – PolarisDMD (Ages 4-7)
36 Figure 11: Catena for DMD (October 6, 2020): Phase III – SIDEROS (w/Glucocorticoid Steroids)
37 Figure 12: Key upcoming events in DMD
38 Figure 13: Probability of success in the DMD pipeline
43 Figure 14: Clinical trials in DMD
43 Figure 15: Top 10 drugs for clinical trials in DMD
44 Figure 16: Top 10 companies for clinical trials in DMD
44 Figure 17: Trial locations in DMD
45 Figure 18: DMD trials status
46 Figure 19: 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: Fordadistrogene Movaparvovec for DMD (December 20, 2021)
22 Table 5: VBP15 for DMD (November 23, 2021)
24 Table 6: Fordadistrogene Movaparvovec for DMD (September 28, 2021)
26 Table 7: VBP15 for DMD (June 1, 2021)
28 Table 8: SRP-9001 for DMD (May 18, 2021)
30 Table 9: SGT-001 for DMD (March 15, 2021)
32 Table 10: SRP-9001 for DMD (January 7, 2021)
34 Table 11: Edasalonexent for DMD (October 26, 2020)
35 Table 12: Catena for DMD (October 6, 2020)
41 Table 13: Historical global sales, by drug ($m), 2016–20
42 Table 14: Forecasted global sales, by drug ($m), 2022–26
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