Market Spotlight: Duchenne Muscular Dystrophy (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。
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.
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.