Market Spotlight: Duchenne Muscular Dystrophy (DMD)

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Report
43 Pages
February 2021
Region: Global
Citeline
ID: 4533529

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

The publisher estimates that in 2019, there were 185,900 prevalent cases of Duchenne muscular dystrophy (DMD) in males worldwide, and forecasts that number to increase to 202,400 prevalent cases by 2028.
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 majority of industry-sponsored drugs in active clinical development for DMD are in Phase II, with only one drug 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 oral route, with the remainder being intravenous, intraarterial, subcutaneous, and inhaled formulations. - High-impact upcoming events for drugs in the DMD space comprise topline Phase I/II trial results for SRP-5051; topline Phase II trial results for RG6356 and Translarna; topline Phase IIb trial results for VBP15; topline Phase III trial results for PF-06939926, givinostat, pamrevlumab, and Vyondys 53; and an estimated PDUFA date for AMONDYS 45.
The overall likelihood of approval of a Phase I DMD asset is 20.4%, and the average probability a drug advances from Phase III is 54.5%. Drugs, on average, take 9.2 years from Phase I to approval, compared to 9.1 years in the overall metabolic space.
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 69% of trials in Phase I–II, and only 31% 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 has the highest number of ongoing clinical trials for DMD, with 13 trials.
Sarepta leads the industry sponsors with the highest overall number of clinical trials for DMD, followed by PTC Therapeutics.

OVERVIEWKEY TAKEAWAYSDISEASE BACKGROUND

TREATMENT

Corticosteroids
Genetic therapies
Vitamin D supplement

EPIDEMIOLOGYMARKETED DRUGSPIPELINE DRUGS

RECENT EVENTS AND ANALYST OPINION

RG6356 for DMD (January 7, 2021)
Edasalonexent for DMD (October 26, 2020)
Catena for DMD (October 6, 2020)
SGT-001 for DMD (December 18, 2019)
SGT-001 for DMD (November 12, 2019)
Vyondys 53 for DMD (August 19, 2019)

KEY UPCOMING EVENTS

KEY REGULATORY EVENTS

Viltepso Is Second Exon 53-Skipping Therapy Approved For DMD
Improved EU Patient Access & Reimbursement On The Cards For Translarna
Another Setback For Santhera’s Idebenone DMD Hopes

PROBABILITY OF SUCCESS

LICENSING AND ASSET ACQUISITION DEALS

Sarepta And Genevant Collaborate For LNP Gene Editing Candidates
Lilly Teams With Precision On DMD, Genetic Diseases
Ultragenyx Enters Duchenne Gene Therapy Space In $295m Deal With Solid Biosciences
Santhera Doubles Its DMD Portfolio By Taking Option On Vamorolone
Sarepta Licenses Hansa Candidate As DMD/LGMD Pre-Treatment
Sarepta Partners With Selecta And Codiak On Gene Therapies

REVENUE OPPORTUNITY

CLINICAL TRIAL LANDSCAPE

Sponsors by status
Sponsors by phase
Recent events

BIBLIOGRAPHY

Prescription information

APPENDIX

LIST OF FIGURES

Figure 1: Trends in prevalent cases of DMD, 2019–28
Figure 2: Overview of pipeline drugs for DMD in the US
Figure 3: Pipeline drugs for DMD, by company
Figure 4: Pipeline drugs for DMD, by drug type
Figure 5: Pipeline drugs for DMD, by classification
Figure 6: RG6356 for DMD (January 7, 2021): Phase II - Study 102
Figure 7: Edasalonexent for DMD (October 26, 2020): Phase III - PolarisDMD (Ages 4-7)
Figure 8: Catena for DMD (October 6, 2020): Phase III - SIDEROS (w/Glucocorticoid Steroids)
Figure 9: Key upcoming events in DMD
Figure 10: Probability of success in the DMD pipeline
Figure 11: Clinical trials in DMD
Figure 12: Top 10 drugs for clinical trials in DMD
Figure 13: Top 10 companies for clinical trials in DMD
Figure 14: Trial locations in DMD
Figure 15: DMD trials status
Figure 16: DMD trials sponsors, by phase

LIST OF TABLES

Table 1: Prevalent cases of DMD, 2019–28
Table 2: Marketed drugs for DMD
Table 3: Pipeline drugs for DMD in the US
Table 4: RG6356 for DMD (January 7, 2021)
Table 5: Edasalonexent for DMD (October 26, 2020)
Table 6: Catena for DMD (October 6, 2020)
Table 7: SGT-001 for DMD (December 18, 2019)
Table 8: SGT-001 for DMD (November 12, 2019)
Table 9: Vyondys 53 for DMD (August 19, 2019)
Table 10: Historical global sales, by drug ($m), 2015–19
Table 11: Forecasted global sales, by drug ($m), 2021–25

Table of Contents

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