Market Spotlight: Huntington's Disease
|出版日期||內容資訊||英文 29 Pages
|焦點市場分析:亨丁頓舞蹈症 Market Spotlight: Huntington's Disease|
|出版日期: 2020年08月14日||內容資訊: 英文 29 Pages||
據估計，2017年全球30歲以上人口中患有亨廷頓舞蹈病的人數為159,410，預計到2026年將增加到184,950。就各地區別來看，北美患病人數最多，而亞洲患病人數最少。臨床開發中的大多數藥物都處於II期，其中兩種藥物處於III期。目標包括亨廷頓蛋白，囊泡單胺轉運蛋白，p38 MAP激酶，SIRT1，血管加壓素受體，semaphorin 4D / CD100，芳基烴受體，NMDA谷氨酸受體，PPARδ，PPARγ等。大多數線藥物是口服的，其他包括鞘內，靜脈內，腦室內和腦室內產品。美國是區域臨床試驗的世界領導者，英國位居歐盟之首。
Huntington's disease (HD) is a rare disorder of the central nervous system (CNS) which causes the degeneration of nerve cells. It is an autosomal dominant disease characterized by involuntary choreatic movements, psychiatric and behavioral disturbances, and dementia. The mean age of symptom onset is 30-50 years. HD is caused by the presence of >40 cytosine-adenine-guanine (CAG) repeats within the Huntingtin gene. The length of the CAG repeats is inversely associated with the age of disease onset - the longer the repeat, the earlier the onset. In the case of juvenile HD (JHD), the length of the repeat is >55 and the disease's symptoms are seen in patients aged <20 years. Learning disabilities and behavioral disturbances are the first symptoms in patients with JHD.
Datamonitor Healthcare estimates that in 2019, there were 77,200 prevalent cases of Huntington's disease (HD) in adults aged 30 years and older worldwide, and forecasts that number to increase to 87,700 prevalent cases by 2028.
Northern America is estimated to have the highest disease prevalence (0.0073%), while Asia has the lowest prevalence (0.0004%).
Teva's Austedo and Bausch Health's Xenazine, which target vesicular monamine transporters, are the only marketed drugs for HD. These drugs are administered via the oral route.
The majority of industry-sponsored drugs in active clinical development for HD are in Phase II, with two drugs in Phase III. Therapies in active clinical development for HD focus on targets such as huntingtin, vesicular monamine transporters, p38 MAP kinase, SIRT1, vasopressin receptors, semaphorin 4D/CD100, NMDA glutamate receptor, PPAR delta, and PPAR gamma. The majority of the pipeline drugs are administered via the oral route, with the remainder being intrathecal, intravenous, and intracerebral/cerebroventricular formulations.
High-impact upcoming events for drugs in the HD space comprise topline Phase Ib/IIa trial results for WVE-120101, topline Phase I/II trial results for AMT-130, topline Phase IIa trial results for ANX-005, and topline Phase III trial results for Ingrezza and tominersen.
The overall likelihood of approval of a Phase I neurodegenerative asset is 7.9%, and the average probability a drug advances from Phase III is 49.5%. Drugs, on average, take 10.9 years from Phase I to approval, compared to 9.8 years in the overall neurology space.
The distribution of clinical trials across Phase I-IV indicates that the majority of trials for HD have been in the early and midphases of development, with 77% of trials in Phase I-II, and only 23% in Phase III-IV.
The US has a substantial lead in the number of HD clinical trials globally, while the UK leads the major European markets. Clinical trial activity in the HD space is dominated by completed trials. Pfizer and Teva have the highest number of completed clinical trials for HD, with 14 trials each.
Pfizer and Teva lead industry sponsors with the highest overall number of clinical trials for HD.