Market Spotlight: Duchenne Muscular Dystrophy (DMD)
Market Spotlight: Duchenne Muscular Dystrophy (DMD)
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
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.
- 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.
- 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 largest proportion of industry-sponsored drugs in active clinical development for DMD are in Phase II, with only 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 I/II, Phase II, Phase IIb, and Phase III trial results, an estimated PDUFA date for an NDA, and an expected option exercise decision.
- The overall likelihood of approval of a Phase I DMD asset is 21.8%, and the average probability a drug advances from Phase III is 62.5%. Drugs, on average, take 8.6 years from Phase I to approval, compared to 8.9 years in the overall metabolic space.
- There have been 21 licensing and asset acquisition deals involving DMD drugs during 2015–20, seven 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 71% of trials in Phase I–II, and only 29% 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 Therapeutics has the highest number of ongoing clinical trials for DMD, with 10 trials.
- Sarepta Therapeutics leads industry sponsors with the highest number of clinical trials for DMD, followed by PTC Therapeutics
7 KEY TAKEAWAYS
8 DISEASE BACKGROUND
9 Genetic therapies
9 Vitamin D supplement
12 MARKETED DRUGS
14 PIPELINE DRUGS
20 RECENT EVENTS AND ANALYST OPINION
20 SGT-001 for DMD (December 18, 2019)
21 SGT-001 for DMD (November 12, 2019)
22 Vyondys 53 for DMD (August 19, 2019)
24 CAP-1002 for DMD (July 15, 2019)
25 CPF-06939926 for DMD (June 28, 2019)
28 SingleCut CRISPR-Cas9 Program (Exonics) for DMD (June 6, 2019)
29 AT702 for DMD (April 8, 2019)
30 Casimersen for DMD (March 28, 2019)
31 Catena for DMD (February 25, 2019)
33 SGT-001 for DMD (February 7, 2019)
34 Emflaza for DMD (January 7, 2019)
35 CAP-1002 for DMD (December 21, 2018)
36 PF-06252616 for DMD (August 30, 2018)
38 Multiple Drugs for DMD (August 30, 2018)
40 KEY UPCOMING EVENTS
41 KEY REGULATORY EVENTS
41 For Sarepta, Another Exon-Skipping Drug
41 Surprise As FDA Approves Sarepta’s Duchenne Drug Vyondys 53
41 Adverse Event Concerns With Solid Biosciences’ Candidate DMD Gene Therapy
41 Sarepta’s Vyondys 53 Whiff Looks Costly As Nippon Shinyaku Submits Viltolarsen
42 Sarepta Surprised By CRL For Exon 53-Skipping DMD Drug Golodirsen
42 PTC To Fight EMA Translarna No
43 PROBABILITY OF SUCCESS
44 LICENSING AND ASSET ACQUISITION DEALS
44 Sarepta Secures $1.15bn From Roche In Ex-US DMD Gene Therapy Deal
44 Astellas To Pay $3bn For Gene Therapy Company Audentes
45 Vertex Gene-Editing Play Includes Exonics Buy, Expanded Tie-Up With CRISPR
45 Audentes Gains License To NCH’s DMD, DM1 Candidates
46 PARENT PATENTS
47 REVENUE OPPORTUNITY
48 CLINICAL TRIAL LANDSCAPE
49 Sponsors by status
50 Sponsors by phase
51 Recent events
54 Prescription information
LIST OF FIGURES
11 Figure 1: Trends in prevalent cases of DMD, 2017–26
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
25 Figure 6: CAP-1002 for DMD (July 15, 2019): Phase II – HOPE-2
31 Figure 7: Casimersen for DMD (March 28, 2019): Phase III – ESSENCE (Study 4045-301; Exon 45 & 53)
33 Figure 8: Catena for DMD (February 25, 2019): Phase III – SYROS
38 Figure 9: PF-06252616 for DMD (August 30, 2018): Phase II – PK and PD Study
40 Figure 10: Key upcoming events in DMD
43 Figure 11: Probability of success in the DMD pipeline
44 Figure 12: Licensing and asset acquisition deals in DMD, 2015–20
46 Figure 13: Parent patents in DMD
48 Figure 14: Clinical trials in DMD
48 Figure 15: Top 10 drugs for clinical trials in DMD
49 Figure 16: Top 10 companies for clinical trials in DMD
49 Figure 17: Trial locations in DMD
50 Figure 18: DMD trials status
51 Figure 19: DMD trials sponsors, by phase
LIST OF TABLES
10 Table 1: Prevalent cases of DMD, 2017–26
13 Table 2: Marketed drugs for DMD
16 Table 3: Pipeline drugs for DMD in the US
20 Table 4: SGT-001 for DMD (December 18, 2019)
21 Table 5: SGT-001 for DMD (November 12, 2019)
23 Table 6: Vyondys 53 for DMD (August 19, 2019)
24 Table 7: CAP-1002 for DMD (July 15, 2019)
26 Table 8: PF-06939926 for DMD (June 28, 2019)
28 Table 9: SingleCut CRISPR-Cas9 Program (Exonics) for DMD (June 6, 2019)
29 Table 10: AT702 for DMD (April 8, 2019)
30 Table 11: Casimersen for DMD (March 28, 2019)
32 Table 12: Catena for DMD (February 25, 2019)
34 Table 13: SGT-001 for DMD (February 7, 2019)
35 Table 14: Emflaza for DMD (January 7, 2019)
36 Table 15: CAP-1002 for DMD (December 21, 2018)
37 Table 16: PF-06252616 for DMD (August 30, 2018)
38 Table 17: Multiple Drugs for DMD (August 30, 2018)
47 Table 18: Historical global sales, by drug ($m), 2014–18
47 Table 19: Forecasted global sales, by drug ($m), 2020–24
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