DUBLIN–(BUSINESS WIRE)–The “Market Spotlight: Duchenne Muscular Dystrophy (DMD)” report has been added to ResearchAndMarkets.com’s offering.
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.
Key Topics Covered:
OVERVIEW
KEY TAKEAWAYS
DISEASE BACKGROUND
TREATMENT
- Corticosteroids
- Genetic therapies
- Vitamin D supplement
EPIDEMIOLOGY
MARKETED DRUGS
PIPELINE 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
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