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市場調查報告書

關注市場分析:裘馨氏肌肉萎縮症 (DMD)

Market Spotlight: Duchenne Muscular Dystrophy (DMD)

出版商 Datamonitor Healthcare 商品編碼 594577
出版日期 內容資訊 英文 61 Pages
商品交期: 最快1-2個工作天內
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關注市場分析:裘馨氏肌肉萎縮症 (DMD) Market Spotlight: Duchenne Muscular Dystrophy (DMD)
出版日期: 2019年11月15日內容資訊: 英文 61 Pages
簡介

本報告提供裘馨氏肌肉萎縮症 (DMD)的治療藥市場相關分析,疾病概要和患者數的變化、預測,目前的主要的治療方法,法規環境和近幾年主要的事件,現在開發中臨床實驗的進展與上市計劃,主要的治療藥的市場趨勢預測 (今後10年份),資本交易的動向等調查。

分析的要點

疾病的背景情況

治療方法

  • 皮質類固醇
  • 基因治療
  • 維他命D供給

流行病學

已上市醫藥品

  • 各國的認證情形

開發平台上的醫藥品

將產生的主要事件

法規相關的主要事件 (共6件)

授權合約、資產收購交易 (共4件)

母體專利

商機

臨床實驗形勢

  • 贊助商數:各狀態
  • 贊助商數:各相位
  • 近幾年趨勢

相關分析

  • 處方藥的資訊

附錄

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目錄
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 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.

TABLE OF CONTENTS

OVERVIEW

KEY TAKEAWAYS

DISEASE BACKGROUND

TREATMENT

  • Corticosteroids
  • Genetic therapies
  • Vitamin D supplement

EPIDEMIOLOGY

MARKETED DRUGS

PIPELINE DRUGS

RECENT EVENTS AND ANALYST OPINION

  • Golodirsen 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)
  • 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)

KEY UPCOMING EVENTS

KEY REGULATORY EVENTS

  • Sarepta's Vyondys 53 Whiff Looks Costly As Nippon Shinyaku Submits Viltolarsen
  • Sarepta Surprised By CRL For Exon 53-Skipping DMD Drug Golodirsen
  • PTC To Fight EMA Translarna No

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: Golodirsen for DMD (August 19, 2019)
  • Table 5: CAP-1002 for DMD (July 15, 2019)
  • Table 6: PF-06939926 for DMD (June 28, 2019)
  • Table 7: SingleCut CRISPR-Cas9 Program (Exonics) for DMD (June 6, 2019)
  • Table 8: AT702 for DMD (April 8, 2019)
  • Table 9: Casimersen for DMD (March 28, 2019)
  • Table 10: Catena for DMD (February 25, 2019)
  • Table 11: SGT-001 for DMD (February 7, 2019)
  • Table 12: Emflaza for DMD (January 7, 2019)
  • Table 13: CAP-1002 for DMD (December 21, 2018)
  • Table 14: PF-06252616 for DMD (August 30, 2018)
  • Table 15: Multiple Drugs for DMD (August 30, 2018)
  • Table 16: Ezutromid for DMD (June 27, 2018)
  • Table 17: Viltolarsen for DMD (June 27, 2018)
  • Table 18: SRP-9001 for DMD (June 19, 2018)
  • Table 19: SGT-001 for DMD (June 18, 2018)
  • Table 20: Historical global sales, by drug ($m), 2014-18
  • Table 21: Forecasted global sales, by drug ($m), 2019-23
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