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 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.
Key Takeaways
- Datamonitor Healthcare estimates that in 2016, there were 179,200 prevalent cases of Duchenne muscular dystrophy in males worldwide, and forecasts that number to increase to 196,200 prevalent cases by 2025.
- Marketed drugs for Duchenne muscular dystrophy include the FDA-approved drugs Emflaza (deflazacort), a glucocorticoid with anti-inflammatory and immunosuppressant properties, and Exondys 51 (eteplirsen), 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 (ataluren), which acts by targeting premature nonsense mutations. These marketed drugs are administered via the oral route, except for Exondys 51, which is available as an intravenous formulation.
- The majority of industry-sponsored drugs in active clinical development for Duchenne muscular dystrophy are in Phase II. Therapies in mid- and late-stage development for Duchenne muscular dystrophy focus on a wide variety of targets. The majority 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 Duchenne muscular dystrophy space comprise an expected CHMP opinion for Exondys 51, topline Phase III trial results for Exondys 51, Catena, and edasalonexent, and topline Phase II trial results for Synacthen Depot.
- There have been 18 licensing and asset acquisition deals involving Duchenne muscular dystrophy drugs during 2013–18, eight of which occurred in 2017. The $562m license and collaboration agreement between Sarepta Therapeutics and Summit Therapeutics for European rights to Summit’s utrophin modulator pipeline for the treatment of Duchenne muscular dystrophy was the largest deal.
- The distribution of clinical trials across Phase I–IV indicates that the majority of trials have been in the early and mid-phases of development, with approximately 72% of trials in Phase I–II, and only 28% in Phase III–IV.
- The US has a substantial lead in the number of Duchenne muscular dystrophy clinical trials globally. The UK leads the major EU markets, while Israel has the top spot in Asia.
- Clinical trial activity in the Duchenne muscular dystrophy space is dominated by ongoing trials. Sarepta Therapeutics has the highest number of ongoing trials for Duchenne muscular dystrophy, with eight trials.
- PTC Therapeutics leads industry sponsors with the highest number of clinical trials for Duchenne muscular dystrophy, 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
14 MARKETED DRUGS
16 Approvals by country
18 PIPELINE DRUGS
25 RECENT EVENTS AND ANALYST OPINION
25 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)
42 Translarna for DMD (September 28, 2017)
44 Translarna for DMD (September 26, 2017)
45 Golodirsen for DMD (September 6, 2017)
47 Golodirsen for DMD (May 18, 2017)
49 SRP-4045 for DMD (May 18, 2017)
50 Translarna for DMD (March 2, 2017)
52 Edasalonexent for DMD (January 31, 2017)
55 KEY UPCOMING EVENTS
56 KEY REGULATORY EVENTS
56 Not The End For Exondys In EU, Says Sarepta
56 Sarepta Should Gain Clean Slate With CBER Review of DMD Gene Therapy
56 Sarepta Will Challenge Expected No From EMA On Exondys 51
56 Sarepta CEO Vows To Get Exondys 51 Into EU Despite CHMP Negative Trend Vote
56 Definitive Data Hopes Keep Santhera’s Duchenne Drug Alive in UK Despite EMA No
56 With Back-To-Back FDA Holds, Solid Hopes To Regain Footing For Duchenne Candidate
57 Refining A Controversial Endpoint: Sarepta Banking On Dystrophin Levels For Golodirsen Approval In DMD
57 PTC’s Ataluren and Accelerated Approval: Is Eteplirsen A Precedent After All?
57 Exondys Revisited? Translarna Brings Efficacy Woes Into US Panel Review
57 Before Price Controversy, Emflaza Got Easy US Nod With Old But Good Enough Data
58 PROBABILITY OF SUCCESS
59 LICENSING AND ASSET ACQUISITION DEALS
59 Sarepta Options Duke’s CRISPR/Cas9 IP For DMD
59 Lucky No. 35? Sarepta And BioMarin Settle Global Patent Disputes
60 Bristol Licenses Phase II DMD Candidate To Roche
60 Sarepta Snaps Up Gene Therapy Approaches to DMD
61 PARENT PATENTS
62 REVENUE OPPORTUNITY
63 CLINICAL TRIAL LANDSCAPE
64 Sponsors by status
65 Sponsors by phase
66 Recent events
66 Sarepta Commits To Rapid, Thorough Pivotal Study For DMD Gene Therapy Based On Functional Improvements
66 In DMD, Summit Falls Well Short Of Peak It Hoped For
66 Sarepta Outlines A Fast Path Forward For Its DMD Gene Therapy
67 Pfizer Advances Duchenne Drug As It Prioritizes Gene Therapy
67 Summit DMD Data Intrigue But Longer-Term Results Awaited
67 Capricor’s DMD Cell Therapy Has HOPE, But Market Is Unsure
67 Confident Catabasis Takes Positives From Failed Duchenne Trial Into Phase III
67 Lost $700m Opportunity As PTC Bids Farewell to Translarna in CF
69 BIBLIOGRAPHY
69 Prescription information
70 APPENDIX
LIST OF FIGURES
13 Figure 1: Trends in prevalent cases of Duchenne muscular dystrophy, 2017–26
18 Figure 2: Overview of pipeline drugs for Duchenne muscular dystrophy in the US
18 Figure 3: Pipeline drugs for Duchenne muscular dystrophy, by company
19 Figure 4: Pipeline drugs for Duchenne muscular dystrophy, by drug type
19 Figure 5: Pipeline drugs for Duchenne muscular dystrophy, by classifications
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)
42 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)
54 Figure 13: Edasalonexent for DMD (January 31, 2017): Phase I/II – MoveDMD
55 Figure 14: Key upcoming events in Duchenne muscular dystrophy
58 Figure 15: Probability of success in the Duchenne muscular dystrophy pipeline
59 Figure 16: Licensing and asset acquisition deals in Duchenne muscular dystrophy, 2013–18
61 Figure 17: Parent patents in Duchenne muscular dystrophy
63 Figure 18: Clinical trials in Duchenne muscular dystrophy
63 Figure 19: Top 10 drugs for clinical trials in Duchenne muscular dystrophy
64 Figure 20: Top 10 companies for clinical trials in Duchenne muscular dystrophy
64 Figure 21: Trial locations in Duchenne muscular dystrophy
65 Figure 22: Duchenne muscular dystrophy trials status
LIST OF TABLES
66 Figure 23: Duchenne muscular dystrophy trials sponsors, by phase
12 Table 1: Prevalent cases of Duchenne muscular dystrophy, 2017–26
15 Table 2: Marketed drugs for Duchenne muscular dystrophy
17 Table 3: Approvals by country for Duchenne muscular dystrophy
20 Table 4: Pipeline drugs for Duchenne muscular dystrophy in the US
25 Table 5: PF-06252616 for DMD (August 30, 2018)
27 Table 6: AAV1-FS344 for DMD (August 30, 2018)
28 Table 7: RG6206 for DMD (August 30, 2018)
30 Table 8: Ezutromid for DMD (June 27, 2018)
31 Table 9: NS-065/NCNP-01 for DMD (June 27, 2018)
33 Table 10: Microdystrophin gene therapy program (NCH) for DMD (June 19, 2018)
35 Table 11: SGT-001 for DMD (June 18, 2018)
36 Table 12: SGT-001 for DMD (March 14, 2018)
37 Table 13: Golodirsen for DMD (March 12, 2018)
38 Table 14: Translarna for DMD (February 20, 2018)
39 Table 15: Ezutromid for DMD (January 25, 2018)
40 Table 16: Translarna for DMD (October 25, 2017)
41 Table 17: Edasalonexent for DMD (October 4, 2017)
43 Table 18: Translarna for DMD (September 28, 2017)
44 Table 19: Translarna for DMD (September 26, 2017)
46 Table 20: Golodirsen for DMD (September 6, 2017)
48 Table 21: Golodirsen for DMD (May 18, 2017)
49 Table 22: SRP-4045 for DMD (May 18, 2017)
51 Table 23: Translarna for DMD (March 2, 2017)
52 Table 24: Edasalonexent for DMD (January 31, 2017)
62 Table 25: Historical global sales, by drug ($m), 2013–17
62 Table 26: Forecasted global sales, by drug ($m), 2018–22
© Informa UK Ltd. This document is a licensed product and is not to be reproduced or redistributed
Do you have a subscription to Datamonitor Healthcare, Biomedtracker or Meddevicetracker? You may already have access to these reports, contact your account manager or email pharma@informabi.com for further help or assistance.
Sign up to the