關注市場分析:裘馨氏肌肉萎縮症 (DMD)

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

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 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.

TABLE OF CONTENTS

CONTENTS

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

LIST OF FIGURES

• Figure 1: Trends in prevalent cases of DMD, 2019-28
• 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: RG6356 for DMD (January 7, 2021): Phase II - Study 102
• Figure 7: Edasalonexent for DMD (October 26, 2020): Phase III - PolarisDMD (Ages 4-7)
• Figure 8: Catena for DMD (October 6, 2020): Phase III - SIDEROS (w/Glucocorticoid Steroids)
• Figure 9: Key upcoming events in DMD
• Figure 10: Probability of success in the DMD pipeline
• Figure 11: Clinical trials in DMD
• Figure 12: Top 10 drugs for clinical trials in DMD
• Figure 13: Top 10 companies for clinical trials in DMD
• Figure 14: Trial locations in DMD
• Figure 15: DMD trials status
• Figure 16: DMD trials sponsors, by phase

LIST OF TABLES

• Table 1: Prevalent cases of DMD, 2019-28
• Table 2: Marketed drugs for DMD
• Table 3: Pipeline drugs for DMD in the US
• Table 4: RG6356 for DMD (January 7, 2021)
• Table 5: Edasalonexent for DMD (October 26, 2020)
• Table 6: Catena for DMD (October 6, 2020)
• Table 7: SGT-001 for DMD (December 18, 2019)
• Table 8: SGT-001 for DMD (November 12, 2019)
• Table 9: Vyondys 53 for DMD (August 19, 2019)
• Table 10: Historical global sales, by drug ($m), 2015-19
• Table 11: Forecasted global sales, by drug ($m), 2021-25