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 report 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. -
Marketed drugs for DMD include the FDA-approved drugs Emflaza, a
glucocorticoid with anti-inflammatory and immunosuppressant
properties, and Exondys 51, 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,
which acts by targeting premature nonsense mutations. Emflaza and
Translarna are administered via the oral route, while Exondys 51 is
available as an intravenous formulation. -
The largest proportion of industry-sponsored drugs in active clinical
development for DMD are in Phase II. Therapies in mid- and late-stage
development for DMD focus on a wide variety of targets. The largest
proportion of pipeline drugs in mid- and late-stage development are
administered orally, with the remainder being intravenous,
intramuscular, intraarterial, and subcutaneous formulations. -
High-impact upcoming events for drugs in the DMD space comprise
topline Phase III, Phase II, and Phase I/II trial results, expected
PDUFA dates for NDA/BLA, and an estimated supplemental CHMP opinion. -
The overall likelihood of approval of a Phase I single-gene disorders
(non-inborn errors of metabolism) asset is 25.8%, and the average
probability a drug advances from Phase III is 66.7%. Drugs, on
average, take 7.4 years from Phase I to approval, compared to 8.9
years in the overall metabolic space. -
There have been 20 licensing and asset acquisition deals involving DMD
drugs during 2014-19, eight of which occurred in 2017. The $562m
license and collaboration agreement between Sarepta Therapeutics and
Summit Therapeutics, for European rights to Summit Therapeutics’
utrophin modulator pipeline for the treatment of DMD, was the largest
deal during the period. -
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 72% of trials in Phase I-II, and only 28% in Phase
III-IV. -
The US has a substantial lead in the number of DMD clinical trials
globally. The UK leads the major EU markets, while Israel has the top
spot in Asia. -
Sarepta Therapeutics has the highest number of ongoing trials for DMD,
with 10 trials. PTC Therapeutics leads industry sponsors with the
highest number of clinical trials for DMD, followed by Sarepta
Therapeutics and BioMarin.
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
Emflaza for DMD (January 7, 2019)
CAP-1002 for DMD (December 21, 2018)
PF-06252616 for DMD (August 30, 2018)
AAV1-FS344 for DMD (August 30, 2018)
RG6206 for DMD (August 30, 2018)
Ezutromid for DMD (June 27, 2018)
NS-065/NCNP-01 for DMD (June 27, 2018)
Microdystrophin Gene Therapy Program (NCH) for DMD (June 19, 2018)
SGT-001 for DMD (June 18, 2018)
SGT-001 for DMD (March 14, 2018)
Golodirsen for DMD (March 12, 2018)
Translarna for DMD (February 20, 2018)
Ezutromid for DMD (January 25, 2018)
Translarna for DMD (October 25, 2017)
Edasalonexent for DMD (October 4, 2017)
Translarna for DMD (September 28, 2017)
Translarna for DMD (September 26, 2017)
Golodirsen for DMD (September 6, 2017)
KEY UPCOMING EVENTS
KEY REGULATORY EVENTS
Not The End For Exondys In EU, Says Sarepta
Sarepta Should Gain Clean Slate With CBER Review of DMD Gene Therapy
Sarepta Will Challenge Expected No From EMA On Exondys 51
Sarepta CEO Vows To Get Exondys 51 Into EU Despite CHMP Negative Trend
Vote
Definitive Data Hopes Keep Santhera’s Duchenne Drug Alive in UK Despite
EMA No
With Back-To-Back FDA Holds, Solid Hopes To Regain Footing For Duchenne
Candidate
Refining A Controversial Endpoint: Sarepta Banking On Dystrophin Levels
For Golodirsen Approval In DMD
PTC’s Ataluren and Accelerated Approval: Is Eteplirsen A Precedent After
All?
PROBABILITY OF SUCCESS
LICENSING AND ASSET ACQUISITION DEALS
Santhera To Snap Up Second DMD Drug As Idorsia Climbs Aboard
PARENT PATENTS
REVENUE OPPORTUNITY
CLINICAL TRIAL LANDSCAPE
Sponsors by status
Sponsors by phase
Recent events
Sarepta Commits To Rapid, Thorough Pivotal Study For DMD Gene Therapy
Based On Functional Improvements
In DMD, Summit Falls Well Short Of Peak It Hoped For
Sarepta Outlines A Fast Path Forward For Its DMD Gene Therapy
Pfizer Advances Duchenne Drug As It Prioritizes Gene Therapy
BIBLIOGRAPHY
Prescription information
APPENDIX
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Related
Topics: Central
Nervous System Drugs , Musculoskeletal
Disorders Drugs