【文　摘】澳大利亚治疗药物管理局已经授予Daval国际公司的皮下给药山羊血清产品Aimspro（Ⅰ）罕见病药地位，以治疗Krabbe疾病（也被称为球状细胞脑白质营养不良）。
      
ＡＩＭＳＰＲＯ被澳授予孤儿药地位
      
英国一家生物技术公司达瓦尔国际有限公司日前宣布，澳大利亚治疗用品管理局（简称ＴＧＡ）已通知该公司，其创新型消炎剂ＡＩＭＳＰＲＯ已获治疗肌萎缩侧索硬化症的罕见药地位。

ＡＩＭＳＰＲＯ是源自并产于澳大利亚的新一代生物药物。它来自超免疫山羊血清，目前正在欧洲进行晚期硬皮病和膀胱功能障碍的继发进展型多发性硬化症的第二阶段试验。两项试验预计于２０１０年第一季度得出报告。长期积累的更多有关ＡＩＭＳＰＲＯ的开放标记经验，已经促使达瓦尔公司考虑对粘连性蛛网膜炎、难治重症肌无力和银屑病关节炎进行验证研究。

Aimspro®

AIMSPRO is composed of a set of peptides which act to stimulate the
release and regulation of a molecular cascade that modulates the
Hypothalamo-Pituitary-Adrenal (HPA) axis. In addition, AIMSPRO has
powerful anti-inflammatory properties as it contains cytokines that induce
a predominantly TH-2 anti-inflammatory profile in the recipient.

It has become apparent that some of the peptides in AIMSPRO are regulated
and bound by specific carrier molecules that may regulate the action and
release of their ligand and are probably necessary for the appropriate
regulation and pharmacokinetics of the molecular machinery. The carrier
molecules also function as a slow release mechanism, releasing bioactive
components over several days.

As well as a presumed general anti-inflammatory mechanism of action, a
number of "open-label" observations in patients with demyelinating
diseases of the central and / or peripheral nervous system led to the
hypothesis that AIMSPRO may improve the security of axonal conduction by a
modification of voltage gated ion channels. Uncontrolled studies suggest a
lowering of sodium channel triggering voltages - the opposite effect to
that of local anaesthetic medications. This remarkable prospect is to be
confirmed under double-blind "crossover" conditions in the Royal Free
Hospital Bladder Function in Secondary Progressive Multiple Sclerosis
study.

AIMSPRO is delivered by sub-cutaneous injection, the typical regimen being
1.0ml every 4 days. The medication is unlicensed. AIMSPRO is listed on the
"export only" section of the Australian Registry of Therapeutic Goods
(ARTG No. 146040; Product No. 234154).

An Orphan Status Designation has been awarded by the Therapeutic Goods
Administration (TGA) for the treatment of Krabbe Leukodystrophy.

伦敦2009年6月2日电 /美通社亚洲/ -- 英国未公开上市的生物技术公司 Daval International Limited  宣布，澳大利亚治疗用品管理局（Therapeutic Goods Administration，简称 TGA）已通知该公司，其创新型消炎剂 AIMSPRO(R) 已荣获治疗肌萎缩侧索硬化症(AmyotrophicLateral Sclerosis, ALS) 的罕见药地位 (Orphan Status)。
肌萎缩侧索硬化症是运动神经元疾病 (Motor Neuron Disease, MND) 的最常见形式，又称路葛雷克氏症 (Lou Gehrig's)。
AIMSPRO 已获得 TGA 授予的治疗克拉勃脑白质营养不良 (Krabbe Leukodystrophy) 的罕见药地位。Daval 如今正在对多中心安慰剂对照的国际随机临床试验进行评估，以确定在双盲条件下呼吸功能较弱的肌萎缩侧索硬化症晚期患者能否受益于该药物。
AIMSPRO
是源自并生产于澳大利亚的新一代生物医药。它来自于超免疫山羊血清，目前正在欧洲进行晚期硬皮病和膀胱功能障碍的继发进展型多发性硬化的第二阶段试验。两项试验预计于2010年第一季度得出报告。长期积累的更多有关 AIMSPRO 的开放标记经验已经促使 Daval 考虑对粘连性蛛网膜炎、难治重症肌无力和银屑病关节炎进行概念验证 (Proof of Concept) 研究。AIMSPRO 在澳大利亚治疗用品登记表 (Australian Therapeutic Goods Registry) 上被列为“仅限出口”，并由 Biotec Distribution (Wales) Limited 进口到欧洲用于临床试验和“指定病人使用”用途。
该产品的兽药形式 Ceremben(R) 在针对标准竞赛用马和纯种马的极为重要的适应症的研究中已显现功效、安全性和耐受性。澳大利亚监管机构 APVMA 最近授予 Ceremben 一项研究许可证，这将使马的现场验证试验得以迅速完成。Ceremben 也正在作为一种潜在的犬和猫抗炎药物而进行开发。