Market Spotlight: Fragile X Syndrome
根據需要特殊教育的兒童相關世代調查的結果，男兒4000人，女孩兒8000人有一人，X染色體脆折症候群 (FXS) 發病的可能性。複數的未來潛力治療藥物正在進行臨床實驗中，某些產品已進入II期。還有2015年至2020年之間已達成五項許可協議和資產收購交易，提高了對FXS治療劑開發的期望。就各國來看，美國在藥物開發方面處於領先地位。
本報告提供全球X染色體脆折症候群 (FXS) 治療藥的市場相關分析，彙整疾病概要和治療方法，目前發病情形，主要的已上市/開發平台醫藥品的概要，近來臨床實驗趨勢與今後的預測，近幾年的授權合約·資本交易的動向，專利趨勢，市場規模趨勢預測 (今後5年份)等資訊，為您概述為以下內容。
Fragile X syndrome (FXS) is a genetic disorder leading to various developmental problems ranging from learning disabilities to cognitive impairment. FXS is caused by mutation of the fragile X mental retardation 1 (FMR1) gene, which usually makes a protein known as fragile X mental retardation protein (FMRP). FMRP is required for normal brain development, and individuals with FXS do not make this protein. Individuals who have other fragile X-associated disorders (FXTAS) have mutations in their FMR1 gene, but they generally make some FMRP
Based on cohorts of children with special educational needs, the prevalence of fragile X syndrome (FXS) is estimated to be 1 in 4,000 in males, and 1 in 8,000 in females.
The majority of industry-sponsored drugs in active clinical development for FXS are in Phase II, with two drugs in Phase III. Therapies in active clinical development for FXS focus on targets such as cannabinoid-1/cannabinoid-2 receptor, phosphodiesterase 4, GABA-A receptor, insulin-like growth factor-1 receptor, GABA-B receptor, 11-beta-hydroxylase, 11 betahydroxysteroid dehydrogenase type 1, and potassium channels. The majority of these pipeline drugs are administered via the oral route.
The overall likelihood of approval of a Phase I neurodevelopmental disorders asset is 2.7%, and the average probability a drug advances from Phase III is 20%. Drugs, on average, take 12.4 years from Phase I to approval, compared to 10.0 years in the overall neurology space.
The distribution of clinical trials across Phase I-IV indicates that the majority of trials for FXS 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 FXS clinical trials globally. France and Spain lead the major European markets, while Israel has the top spot in Asia.
Clinical trial activity in the FXS space is dominated by completed trials. Seaside Therapeutics and Novartis have the highest number of completed clinical trials for FXS, with four trials each.
Seaside Therapeutics leads industry sponsors with the highest overall number of clinical trials for FXS, followed by Novartis.