麦科奥特1类创新药注射用MT200605亨廷顿舞蹈病获美国FDA孤儿药资格认定
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麦科奥特1类创新药注射用MT200605
亨廷顿舞蹈病获美国FDA孤儿药资格认定
2026年3月13日，陕西麦科奥特医药科技股份有限公司(以下简称“麦科奥特”)自主研发的1类创新药注射用MT200605，针对亨廷顿舞蹈病（Huntington’s Disease，HD）适应症正式获得美国食品药品监督管理局孤儿药资格认定（Orphan Drug Designation, ODD）。
亨廷顿舞蹈病被称为“史上最残酷遗传病”，是一种常染色体显性遗传的神经退行性罕见病。其病因是HTT基因异常扩增，产生有毒的突变亨廷顿蛋白（mHTT）并沉积于大脑，逐步破坏控制运动、情绪和认知的核心中枢，导致患者肢体失控、认知衰退，给患者及家庭带来沉重身心与经济负担。目前全球尚无根治药物，现有治疗仅能缓解症状，无法阻断病情进展，临床需求极为迫切。
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注射用MT200605与现有治疗药物相比
具有显著差异化优势
01
作用机制更具针对性。MT200605可选择性激活TrkB信号通路，同时发挥保护神经组织结构和功能、抗氧化的双重作用，临床前研究已展现出其能够直接减少突变亨廷顿蛋白（mHTT）聚集，从根源上实现“疾病修正”的治疗目标。
02
安全性与成药性更具优势。Ⅰ期临床试验（首次人体试验）数据显示，MT200605静脉制剂在健康受试者中展现出良好的安全性和可预测的药代动力学特征。
03
剂型布局更贴合临床需求。同步开发口服及静脉注射两种制剂，可针对不同病情阶段、不同身体状态的患者提供个性化治疗选择。
孤儿药资格认定是对MT200605临床价值与研发潜力的高度认可，同时也标志着麦科奥特的自主创新能力得到国际权威机构认可。未来，麦科奥特将依托FDA孤儿药认定的政策优势，为全球亨廷顿舞蹈病患者提供安全有效更具针对性的治疗选择。
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Micot’s MT200605 Receives U.S. FDA Orphan Drug Designation for Huntington’s Disease
Xi’an, China — March 13, 2026 — Shaanxi Micot Pharmaceutical Technology Co., Ltd. (“Micot”), an innovative pharmaceutical company focused on developing novel therapeutics for serious diseases, today announced that its investigational therapy MT200605 injection has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) for the treatment of Huntington's Disease.
The designation marks an important milestone for Micot’s neurodegenerative disease pipeline and highlights the therapeutic potential of MT200605 in addressing a significant unmet medical need in rare diseases.
Addressing a Critical Unmet Need
Huntington’s disease is a rare, inherited neurodegenerative disorder caused by a mutation in the HTT gene, leading to the accumulation of toxic mutant huntingtin protein (mHTT) in the brain. The disease progressively impairs motor function, cognition, and emotional regulation, ultimately leading to severe disability and premature death.
With a 50% inheritance risk for the children of affected individuals, Huntington’s disease places a substantial burden on patients, families, and healthcare systems worldwide. Currently available treatments only address symptoms and do not halt disease progression, underscoring the urgent need for disease-modifying therapies.
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Differentiated Mechanism and Development Strategy
MT200605 is a Class 1 innovative chemical drug candidate discovered and developed independently by Micot, with global intellectual property rights owned by the company.
Compared with existing symptomatic therapies, MT200605 is designed to target the disease biology more directly through several differentiating features:
01
Targeted neuroprotective mechanism
MT200605 selectively activates the TrkB signaling pathway, a key regulator of neuronal survival and function. Preclinical studies suggest the compound may reduce aggregation of mutant huntingtin protein (mHTT), potentially protecting neurons and slowing neurodegeneration.
02
Encouraging safety profile
In a completed Phase I first-in-human clinical trial, the intravenous formulation demonstrated favorable safety and tolerability, as well as predictable pharmacokinetics in healthy volunteers.
03
Dual-formulation strategy
Micot is developing both intravenous and oral formulations of MT200605. This strategy aims to support treatment flexibility across different stages of disease and improve long-term patient management.
Strengthening Micot’s Global Innovation Strategy
The ODD designation also represents recognition of Micot’s research and development capabilities and international regulatory standards. The company is committed to expanding its presence in the global innovative drug market while advancing new treatment options for patients with rare diseases.
Looking Ahead：
Micot plans to continue advancing the clinical development of MT200605 through collaborations with global clinical research partners and leading academic institutions. The company aims to accelerate the development of both the oral and intravenous formulations with the goal of delivering safer, more effective, and targeted therapies for Huntington’s disease patients worldwide.
Micot remains committed to its mission:
“Discovering and developing innovative medicines to address unmet medical needs and improve global health.”
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