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Market Spotlight: Duchenne Muscular Dystrophy (DMD)

  • ID: 4533529
  • Report
  • 61 pages
  • Datamonitor Healthcare
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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 author 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, with two drugs 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 intravenous or oral routes, with the remainder being intramuscular, intraarterial, and subcutaneous formulations.
  • High-impact upcoming events for drugs in the DMD space comprise topline Phase II and Phase III trial results, and an expected PDUFA date for an NDA.
  • The overall likelihood of approval of a Phase I single-gene disorders (non-inborn errors of metabolism) asset is 23.9%, and the average probability a drug advances from Phase III is 66.7%. Drugs, on average, take 7.2 years from Phase I to approval, compared to 8.8 years in the overall metabolic space.
  • There have been 21 licensing and asset acquisition deals involving DMD drugs during 2014–19, eight of which occurred in 2017. The largest deal during the period was the $3,520m licensing agreement between Vertex and CRISPR Therapeutics signed in 2017, as part of which the two companies will discover and develop gene editing therapies for the treatment of DMD and myotonic dystrophy type 1 (DM1).
  • 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 70% of trials in Phase I–II, and only 30% 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 nine trials.
  • PTC Therapeutics leads industry sponsors with the highest number of clinical trials for DMD, followed by Sarepta Therapeutics.
Note: Product cover images may vary from those shown
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OVERVIEW

KEY TAKEAWAYS

DISEASE BACKGROUND

TREATMENT
  • Corticosteroids
  • Genetic therapies
  • Vitamin D supplement
EPIDEMIOLOGY

MARKETED DRUGS

PIPELINE DRUGS

RECENT EVENTS AND ANALYST OPINION
  • 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)
  • Emflaza for DMD (January 7, 2019)
  • CAP-1002 for DMD (December 21, 2018)
  • PF-06252616 for DMD (August 30, 2018)
  • Multiple Drugs for DMD (August 30, 2018)
  • Ezutromid for DMD (June 27, 2018)
  • Viltolarsen for DMD (June 27, 2018)
  • SRP-9001 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)
KEY UPCOMING EVENTS

KEY REGULATORY EVENTS
  • PTC To Fight EMA Translarna No
  • Not The End For Exondys In EU, Says Sarepta
PROBABILITY OF SUCCESS

LICENSING AND ASSET ACQUISITION DEALS
  • Vertex Gene-Editing Play Includes Exonics Buy, Expanded Tie-Up With CRISPR
  • Audentes Gains License To NCH's DMD, DM1 Candidates
  • 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
BIBLIOGRAPHY
  • Prescription information
APPENDIX

LIST OF FIGURES
Figure 1: Trends in prevalent cases of DMD, 2017–26
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: PF-06252616 for DMD (August 30, 2018): Phase II - PK and PD Study
Figure 10: Ezutromid for DMD (June 27, 2018): Phase II - PhaseOut DMD (US and EU)
Figure 11: Viltolarsen for DMD (June 27, 2018): Phase II - 201
Figure 12: SRP-9001 for DMD (June 19, 2018): Phase I/IIa - pilot study
Figure 13: Key upcoming events in DMD
Figure 14: Probability of success in the DMD pipeline
Figure 15: Licensing and asset acquisition deals in DMD, 2014–19
Figure 16: Parent patents in DMD
Figure 17: Clinical trials in DMD
Figure 18: Top 10 drugs for clinical trials in DMD
Figure 19: Top 10 companies for clinical trials in DMD
Figure 20: Trial locations in DMD
Figure 21: DMD trials status
Figure 22: DMD trials sponsors, by phase

LIST OF TABLES
Table 1: Prevalent cases of DMD, 2017–26
Table 2: Marketed drugs for DMD
Table 3: Pipeline drugs for DMD in the US
Table 4: CAP-1002 for DMD (July 15, 2019)
Table 5: PF-06939926 for DMD (June 28, 2019)
Table 6: SingleCut CRISPR-Cas9 Program (Exonics) for DMD (June 6, 2019)
Table 7: AT702 for DMD (April 8, 2019)
Table 8: Casimersen for DMD (March 28, 2019)
Table 9: Catena for DMD (February 25, 2019)
Table 10: SGT-001 for DMD (February 7, 2019)
Table 11: Emflaza for DMD (January 7, 2019)
Table 12: CAP-1002 for DMD (December 21, 2018)
Table 13: PF-06252616 for DMD (August 30, 2018)
Table 14: Multiple Drugs for DMD (August 30, 2018)
Table 15: Ezutromid for DMD (June 27, 2018)
Table 16: Viltolarsen for DMD (June 27, 2018)
Table 17: SRP-9001 for DMD (June 19, 2018)
Table 18: SGT-001 for DMD (June 18, 2018)
Table 19: SGT-001 for DMD (March 14, 2018)
Table 20: Golodirsen for DMD (March 12, 2018)
Table 21: Translarna for DMD (February 20, 2018)
Table 22: Historical global sales, by drug ($m), 2014–18
Table 23: Forecasted global sales, by drug ($m), 2019–23
Note: Product cover images may vary from those shown
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