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. Therapies in mid- and late-stage development for DMD focus on a wide variety of targets. The largest proportion of pipeline drugs in mid- and late-stage development are administered orally, with the remainder being intravenous, intramuscular, intraarterial, and subcutaneous formulations.
- High-impact upcoming events for drugs in the DMD space comprise topline Phase III, Phase II, and Phase I/II trial results, expected PDUFA dates for NDA/BLA, and an estimated supplemental CHMP opinion.
- The overall likelihood of approval of a Phase I single-gene disorders (non-inborn errors of metabolism) asset is 25.8%, and the average probability a drug advances from Phase III is 66.7%. Drugs, on average, take 7.4 years from Phase I to approval, compared to 8.9 years in the overall metabolic space.
- There have been 20 licensing and asset acquisition deals involving DMD drugs during 2014–19, eight of which occurred in 2017. The $562m license and collaboration agreement between Sarepta Therapeutics and Summit Therapeutics, for European rights to Summit Therapeutics’ utrophin modulator pipeline for the treatment of DMD, was the largest deal during the period.
- 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 72% of trials in Phase I–II, and only 28% 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 10 trials. PTC Therapeutics leads industry sponsors with the highest number of clinical trials for DMD, followed by Sarepta Therapeutics and BioMarin.
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
23 RECENT EVENTS AND ANALYST OPINION
23 Emflaza for DMD (January 7, 2019)
23 CAP-1002 for DMD (December 21, 2018)
24 PF-06252616 for DMD (August 30, 2018)
26 AAV1-FS344 for DMD (August 30, 2018)
28 RG6206 for DMD (August 30, 2018)
29 Ezutromid for DMD (June 27, 2018)
31 NS-065/NCNP-01 for DMD (June 27, 2018)
33 Microdystrophin Gene Therapy Program (NCH) for DMD (June 19, 2018)
35 SGT-001 for DMD (June 18, 2018)
36 SGT-001 for DMD (March 14, 2018)
37 Golodirsen for DMD (March 12, 2018)
38 Translarna for DMD (February 20, 2018)
38 Ezutromid for DMD (January 25, 2018)
40 Translarna for DMD (October 25, 2017)
41 Edasalonexent for DMD (October 4, 2017)
43 Translarna for DMD (September 28, 2017)
44 Translarna for DMD (September 26, 2017)
46 Golodirsen for DMD (September 6, 2017)
48 KEY UPCOMING EVENTS
49 KEY REGULATORY EVENTS
49 Not The End For Exondys In EU, Says Sarepta
49 Sarepta Should Gain Clean Slate With CBER Review of DMD Gene Therapy
49 Sarepta Will Challenge Expected No From EMA On Exondys 51
49 Sarepta CEO Vows To Get Exondys 51 Into EU Despite CHMP Negative Trend Vote
49 Definitive Data Hopes Keep Santhera’s Duchenne Drug Alive in UK Despite EMA No
49 With Back-To-Back FDA Holds, Solid Hopes To Regain Footing For Duchenne Candidate
50 Refining A Controversial Endpoint: Sarepta Banking On Dystrophin Levels For Golodirsen Approval In DMD
50 PTC’s Ataluren and Accelerated Approval: Is Eteplirsen A Precedent After All?
51 PROBABILITY OF SUCCESS
52 LICENSING AND ASSET ACQUISITION DEALS
52 Santhera To Snap Up Second DMD Drug As Idorsia Climbs Aboard
54 PARENT PATENTS
55 REVENUE OPPORTUNITY
56 CLINICAL TRIAL LANDSCAPE
57 Sponsors by status
58 Sponsors by phase
59 Recent events
59 Sarepta Commits To Rapid, Thorough Pivotal Study For DMD Gene Therapy Based On Functional Improvements
59 In DMD, Summit Falls Well Short Of Peak It Hoped For
59 Sarepta Outlines A Fast Path Forward For Its DMD Gene Therapy
60 Pfizer Advances Duchenne Drug As It Prioritizes Gene Therapy
61 BIBLIOGRAPHY
61 Prescription information
62 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
26 Figure 6: PF-06252616 for DMD (August 30, 2018): Phase II – PK and PD Study
31 Figure 7: Ezutromid for DMD (June 27, 2018): Phase II – PhaseOut DMD (US and EU)
33 Figure 8: NS-065/NCNP-01 for DMD (June 27, 2018): Phase II – 201
35 Figure 9: Microdystrophin gene therapy program (NCH) for DMD (June 19, 2018): Phase I/IIa – pilot study
40 Figure 10: Ezutromid for DMD (January 25, 2018): Phase II – PhaseOut DMD (US and EU)
43 Figure 11: Edasalonexent for DMD (October 4, 2017): Phase I/II – MoveDMD
47 Figure 12: Golodirsen for DMD (September 6, 2017): Phase I/II – 4053-101 (EU)
48 Figure 13: Key upcoming events in DMD
51 Figure 14: Probability of success in the DMD pipeline
52 Figure 15: Licensing and asset acquisition deals in DMD, 2014–19
54 Figure 16: Parent patents in DMD
56 Figure 17: Clinical trials in DMD
56 Figure 18: Top 10 drugs for clinical trials in DMD
57 Figure 19: Top 10 companies for clinical trials in DMD
57 Figure 20: Trial locations in DMD
58 Figure 21: DMD trials status
59 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
23 Table 4: Emflaza for DMD (January 7, 2019)
24 Table 5: CAP-1002 for DMD (December 21, 2018)
25 Table 6: PF-06252616 for DMD (August 30, 2018)
27 Table 7: AAV1-FS344 for DMD (August 30, 2018)
28 Table 8: RG6206 for DMD (August 30, 2018)
30 Table 9: Ezutromid for DMD (June 27, 2018)
31 Table 10: NS-065/NCNP-01 for DMD (June 27, 2018)
33 Table 11: Microdystrophin gene therapy program (NCH) for DMD (June 19, 2018)
35 Table 12: SGT-001 for DMD (June 18, 2018)
36 Table 13: SGT-001 for DMD (March 14, 2018)
37 Table 14: Golodirsen for DMD (March 12, 2018)
38 Table 15: Translarna for DMD (February 20, 2018)
39 Table 16: Ezutromid for DMD (January 25, 2018)
41 Table 17: Translarna for DMD (October 25, 2017)
42 Table 18: Edasalonexent for DMD (October 4, 2017)
43 Table 19: Translarna for DMD (September 28, 2017)
45 Table 20: Translarna for DMD (September 26, 2017)
46 Table 21: Golodirsen for DMD (September 6, 2017)
55 Table 22: Historical global sales, by drug ($m), 2013–17
55 Table 23: Forecasted global sales, by drug ($m), 2019–23
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