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.

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 include US FDA-approved drugs such as 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.

Therapies in mid- and late-stage development for Duchenne muscular dystrophy focus on a variety of targets. The majority of pipeline drugs in mid- and late-stage development for Duchenne muscular dystrophy are administered orally, with the remainder including intravenous, intramuscular, intra-arterial, and subcutaneous formulations.

High-impact upcoming events for drugs in the Duchenne muscular dystrophy space include topline Phase II and Phase III trial results, an estimated PDUFA, and a positive CHMP opinion.

There were 14 licensing and asset acquisition activities involving Duchenne muscular dystrophy drugs during 2012–17, six of which occurred in 2017. The $730m R&D collaboration agreement between Astellas and Mitokyne (now Mitobridge) to discover and develop compounds that target mitochondrial function was the largest deal during 2012–17.

The clinical trials distribution across Phase I–III indicates that the majority of trials for Duchenne muscular dystrophy have been in Phase II development, with 51.1% of trials in Phase II, and only 39.4% and 9.1% trials in Phase III and I, respectively.

The US has a substantial lead in the number of Duchenne muscular dystrophy clinical trials globally. Belgium 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. PTC Therapeutics has the highest number of ongoing and completed clinical trials for Duchenne muscular dystrophy.

PTC Therapeutics leads industry sponsors with the highest number of clinical trials for Duchenne muscular dystrophy, followed by GlaxoSmithKline

 

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Major Table of Contents

1 KEY TAKEAWAYS

2 DISEASE BACKGROUND

3 TREATMENT
4 Corticosteroids
5 Genetic therapies
6 Vitamin D supplement

7 EPIDEMIOLOGY

8 MARKETED DRUGS
9 Approvals by country

10 PIPELINE DRUGS

11 KEY UPCOMING EVENTS

12 KEY REGULATORY EVENTS
13 Exondys Revisited? Translarna Brings Efficacy Woes Into US Panel Review
14 Before Price Controversy, Emflaza Got Easy US Nod With Old But Good Enough Data
15 Sarepta's Exondys 51 (Eteplirsen) Approved After Contentious Internal FDA Debate
16 Marathon Looking To Go The Distance In DMD
17 Scots Hammer PTC's Translarna As EU Reviews Conditional Approval
18 FDA Refuses To File Translarna NDA; PTC Plummets

19 LICENSING AND ASSET ACQUISITION DEALS
20 Lucky No. 35? Sarepta And BioMarin Settle Global Patent Disputes
21 Bristol Licenses Phase II DMD Candidate To Roche
22 Sarepta Snaps Up Gene Therapy Approaches to DMD
23 Sarepta’s $852m Summit Collaboration Could Enable DMD Drug Combinations
24 PARENT PATENTS

25 REVENUE OPPORTUNITY

26 CLINICAL TRIAL LANDSCAPE
27 Sponsors by status
28 Sponsors by phase
29 Recent events

30 BIBLIOGRAPHY
31 Prescription information

32 APPENDIX

LIST OF FIGURES

Figure 1: Trends in prevalent cases of Duchenne muscular dystrophy, 2016–25
Figure 2: Overview of pipeline drugs for Duchenne muscular dystrophy in the US
Figure 3: Pipeline drugs for Duchenne muscular dystrophy, by company
Figure 4: Pipeline drugs for Duchenne muscular dystrophy, by drug type
Figure 5: Pipeline drugs for Duchenne muscular dystrophy, by classifications
Figure 6: Key upcoming events in Duchenne muscular dystrophy
Figure 7: Licensing and asset acquisition deals in disease name, 2012–17
Figure 8: Parent patents in Duchenne muscular dystrophy
Figure 9: Clinical trials in Duchenne muscular dystrophy
Figure 10: Top 10 drugs for clinical trials in Duchenne muscular dystrophy
Figure 11: Top 10 companies for clinical trials in Duchenne muscular dystrophy
Figure 12: Trial locations in Duchenne muscular dystrophy
Figure 13: Duchenne muscular dystrophy trials status
Figure 14: Duchenne muscular dystrophy trials sponsors, by phase

LIST OF TABLES

Table 1: Prevalent cases of Duchenne muscular dystrophy, 2016–25
Table 2: Marketed drugs for Duchenne muscular dystrophy
Table 3: Approvals by country for Duchenne muscular dystrophy
Table 4: Pipeline drugs for Duchenne muscular dystrophy in the US
Table 5: Historical global sales, by drug ($m), 2012–16
Table 6: Forecasted global sales, by drug ($m), 2017–22

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