Product Code: DMKC0181105
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 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 largest proportion 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, 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 expected option exercise decision, an estimated PDUFA date for an NDA, and a CHMP opinion.
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 66.7%. Drugs, on average, take 8.7 years from Phase I to approval, compared to 8.9 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 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 has the highest number of ongoing clinical trials for DMD, with 11 trials.
PTC Therapeutics and Sarepta lead the industry sponsors with the highest overall number of clinical trials for DMD.
TABLE OF CONTENTS
CONTENTS
OVERVIEW
KEY TAKEAWAYS
DISEASE BACKGROUND
TREATMENT
- Corticosteroids
- Genetic therapies
- Vitamin D supplement
EPIDEMIOLOGY
MARKETED DRUGS
PIPELINE DRUGS
RECENT EVENTS AND ANALYST OPINION
- SGT-001 for DMD (December 18, 2019)
- SGT-001 for DMD (November 12, 2019)
- Vyondys 53 for DMD (August 19, 2019)
- CAP-1002 for DMD (July 15, 2019)
- PF-06939926 for DMD (June 28, 2019)
- SingleCut CRISPR-Cas9 Program (Exonics) for DMD (June 6, 2019)
- AT702 for DMD (April 8, 2019)
- Casimersen for DMD (March 28, 2019)
- Catena for DMD (February 25, 2019)
- SGT-001 for DMD (February 7, 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
- For Sarepta, Another Exon-Skipping Drug
- Surprise As FDA Approves Sarepta's Duchenne Drug Vyondys 53
- Adverse Event Concerns With Solid Biosciences' Candidate DMD Gene Therapy
- Sarepta's Vyondys 53 Whiff Looks Costly As Nippon Shinyaku Submits Viltolarsen
- Sarepta Surprised By CRL For Exon 53-Skipping DMD Drug Golodirsen
PROBABILITY OF SUCCESS
LICENSING AND ASSET ACQUISITION DEALS
- Sarepta Licenses Hansa Candidate As DMD/LGMD Pre-Treatment
- Sarepta Partners With Selecta And Codiak On Gene Therapies
- Sarepta Secures $1.15bn From Roche In Ex-US DMD Gene Therapy Deal
- Astellas To Pay $3bn For Gene Therapy Company Audentes
REVENUE OPPORTUNITY
CLINICAL TRIAL LANDSCAPE
- Sponsors by status
- Sponsors by phase
- Recent events
BIBLIOGRAPHY
APPENDIX
LIST OF FIGURES
- Figure 1: Trends in prevalent cases of DMD, 2019-28
- Figure 2: Overview of pipeline drugs for DMD in the US
- Figure 3: Pipeline drugs for DMD, by company
- Figure 4: Pipeline drugs for DMD, by drug type
- Figure 5: Pipeline drugs for DMD, by classification
- Figure 6: CAP-1002 for DMD (July 15, 2019): Phase II - HOPE-2
- Figure 7: Casimersen for DMD (March 28, 2019): Phase III - ESSENCE (Study 4045-301; Exon 45 & 53)
- Figure 8: Catena for DMD (February 25, 2019): Phase III - SYROS
- Figure 9: Key upcoming events in DMD
- Figure 10: Probability of success in the DMD pipeline
- Figure 11: Clinical trials in DMD
- Figure 12: Top 10 drugs for clinical trials in DMD
- Figure 13: Top 10 companies for clinical trials in DMD
- Figure 14: Trial locations in DMD
- Figure 15: DMD trials status
- Figure 16: DMD trials sponsors, by phase
LIST OF TABLES
- Table 1: Prevalent cases of DMD, 2019-28
- Table 2: Marketed drugs for DMD
- Table 3: Pipeline drugs for DMD in the US
- Table 4: SGT-001 for DMD (December 18, 2019)
- Table 5: SGT-001 for DMD (November 12, 2019)
- Table 6: Vyondys 53 for DMD (August 19, 2019)
- Table 7: CAP-1002 for DMD (July 15, 2019)
- Table 8: PF-06939926 for DMD (June 28, 2019)
- Table 9: SingleCut CRISPR-Cas9 Program (Exonics) for DMD (June 6, 2019)
- Table 10: AT702 for DMD (April 8, 2019)
- Table 11: Casimersen for DMD (March 28, 2019)
- Table 12: Catena for DMD (February 25, 2019)
- Table 13: SGT-001 for DMD (February 7, 2019)
- Table 14: Historical global sales, by drug ($m), 2015-19
- Table 15: Forecasted global sales, by drug ($m), 2020-24
