Global Duchenne Muscular Dystrophy (DMD) Market Spotlight 2017-2018 & 2019-2026 - ResearchAndMarkets.com

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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