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 2017, there were 182,100 prevalent cases of Duchenne muscular dystrophy (DMD) in males worldwide, and forecasts that number to increase to 199,100 prevalent cases by 2026.
- Marketed drugs for DMD include the FDA-approved drugs Emflaza, a glucocorticoid with anti-inflammatory and immunosuppressant properties, and Exondys 51, a novel phosphorodiamidate morpholino oligomer designed to induce the skipping of exon 51 in the dystrophin gene. The marketed drugs also include an EU-approved drug, Translarna, which acts by targeting premature nonsense mutations. Emflaza and Translarna are administered via the oral route, while Exondys 51 is available as an intravenous formulation.
- The largest proportion of industry-sponsored drugs in active clinical development for DMD are in Phase II, with two drugs 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 intravenous or oral routes, with the remainder being intramuscular, intraarterial, and subcutaneous formulations.
- High-impact upcoming events for drugs in the DMD space comprise topline Phase II and Phase III trial results, and an expected PDUFA date for an NDA.
- The overall likelihood of approval of a Phase I single-gene disorders (non-inborn errors of metabolism) asset is 23.9%, and the average probability a drug advances from Phase III is 66.7%. Drugs, on average, take 7.2 years from Phase I to approval, compared to 8.8 years in the overall metabolic space.
- There have been 21 licensing and asset acquisition deals involving DMD drugs during 2014–19, eight of which occurred in 2017. The largest deal during the period was the $3,520m licensing agreement between Vertex and CRISPR Therapeutics signed in 2017, as part of which the two companies will discover and develop gene editing therapies for the treatment of DMD and myotonic dystrophy type 1 (DM1).
- 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 70% of trials in Phase I–II, and only 30% in Phase III–IV.
- The US has a substantial lead in the number of DMD clinical trials globally. The UK leads the major EU markets, while Israel has the top spot in Asia.
- Sarepta Therapeutics has the highest number of ongoing trials for DMD, with nine trials.
- PTC Therapeutics leads industry sponsors with the highest number of clinical trials for DMD, followed by Sarepta Therapeutics.
CONTENTS
7 OVERVIEW
8 KEY TAKEAWAYS
9 DISEASE BACKGROUND
10 TREATMENT
10 Corticosteroids
10 Genetic therapies
10 Vitamin D supplement
11 EPIDEMIOLOGY
13 MARKETED DRUGS
15 PIPELINE DRUGS
21 RECENT EVENTS AND ANALYST OPINION
21 Golodirsen for DMD (August 19, 2019)
22 CAP-1002 for DMD (July 15, 2019)
24 PF-06939926 for DMD (June 28, 2019)
27 SingleCut CRISPR-Cas9 Program (Exonics) for DMD (June 6, 2019)
28 AT702 for DMD (April 8, 2019)
29 Casimersen for DMD (March 28, 2019)
30 Catena for DMD (February 25, 2019)
32 SGT-001 for DMD (February 7, 2019)
34 Emflaza for DMD (January 7, 2019)
34 CAP-1002 for DMD (December 21, 2018)
35 PF-06252616 for DMD (August 30, 2018)
37 Multiple Drugs for DMD (August 30, 2018)
38 Ezutromid for DMD (June 27, 2018)
40 Viltolarsen for DMD (June 27, 2018)
42 SRP-9001 for DMD (June 19, 2018)
44 SGT-001 for DMD (June 18, 2018)
46 KEY UPCOMING EVENTS
47 KEY REGULATORY EVENTS
47 Sarepta’s Vyondys 53 Whiff Looks Costly As Nippon Shinyaku Submits Viltolarsen
47 Sarepta Surprised By CRL For Exon 53-Skipping DMD Drug Golodirsen
47 PTC To Fight EMA Translarna No
48 PROBABILITY OF SUCCESS
49 LICENSING AND ASSET ACQUISITION DEALS
49 Vertex Gene-Editing Play Includes Exonics Buy, Expanded Tie-Up With CRISPR
49 Audentes Gains License To NCH’s DMD, DM1 Candidates
50 Santhera To Snap Up Second DMD Drug As Idorsia Climbs Aboard
51 PARENT PATENTS
52 REVENUE OPPORTUNITY
53 CLINICAL TRIAL LANDSCAPE
54 Sponsors by status
55 Sponsors by phase
56 Recent events
59 BIBLIOGRAPHY
59 Prescription information
60 APPENDIX
LIST OF FIGURES
12 Figure 1: Trends in prevalent cases of DMD, 2017–26
15 Figure 2: Overview of pipeline drugs for DMD in the US
15 Figure 3: Pipeline drugs for DMD, by company
16 Figure 4: Pipeline drugs for DMD, by drug type
16 Figure 5: Pipeline drugs for DMD, by classification
24 Figure 6: CAP-1002 for DMD (July 15, 2019): Phase II – HOPE-2
30 Figure 7: Casimersen for DMD (March 28, 2019): Phase III – ESSENCE (Study 4045-301; Exon 45 & 53)
32 Figure 8: Catena for DMD (February 25, 2019): Phase III – SYROS
37 Figure 9: PF-06252616 for DMD (August 30, 2018): Phase II – PK and PD Study
40 Figure 10: Ezutromid for DMD (June 27, 2018): Phase II – PhaseOut DMD (US and EU)
42 Figure 11: Viltolarsen for DMD (June 27, 2018): Phase II – 201
44 Figure 12: SRP-9001 for DMD (June 19, 2018): Phase I/IIa – pilot study
46 Figure 13: Key upcoming events in DMD
48 Figure 14: Probability of success in the DMD pipeline
49 Figure 15: Licensing and asset acquisition deals in DMD, 2014–19
51 Figure 16: Parent patents in DMD
53 Figure 17: Clinical trials in DMD
53 Figure 18: Top 10 drugs for clinical trials in DMD
54 Figure 19: Top 10 companies for clinical trials in DMD
54 Figure 20: Trial locations in DMD
55 Figure 21: DMD trials status
56 Figure 22: DMD trials sponsors, by phase
LIST OF TABLES
11 Table 1: Prevalent cases of DMD, 2017–26
14 Table 2: Marketed drugs for DMD
17 Table 3: Pipeline drugs for DMD in the US
21 Table 4: Golodirsen for DMD (August 19, 2019)
22 Table 5: CAP-1002 for DMD (July 15, 2019)
25 Table 6: PF-06939926 for DMD (June 28, 2019)
27 Table 7: SingleCut CRISPR-Cas9 Program (Exonics) for DMD (June 6, 2019)
28 Table 8: AT702 for DMD (April 8, 2019)
29 Table 9: Casimersen for DMD (March 28, 2019)
31 Table 10: Catena for DMD (February 25, 2019)
33 Table 11: SGT-001 for DMD (February 7, 2019)
34 Table 12: Emflaza for DMD (January 7, 2019)
35 Table 13: CAP-1002 for DMD (December 21, 2018)
36 Table 14: PF-06252616 for DMD (August 30, 2018)
37 Table 15: Multiple Drugs for DMD (August 30, 2018)
39 Table 16: Ezutromid for DMD (June 27, 2018)
40 Table 17: Viltolarsen for DMD (June 27, 2018)
42 Table 18: SRP-9001 for DMD (June 19, 2018)
44 Table 19: SGT-001 for DMD (June 18, 2018)
52 Table 20: Historical global sales, by drug ($m), 2014–18
52 Table 21: Forecasted global sales, by drug ($m), 2019–23
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