LifeMax Receives Orphan Drug Designation From the United States Food & Drug Administration For LM-030 For The Treatment of Netherton Syndrome

LifeMax公司治疗内瑟顿综合征药物LM-030获美国FDA孤儿药认定

2019-06-28 09:07:00 BioSpace

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LifeMax Laboratories, Inc. , a private company focused on treating rare diseases with few or no therapeutic options, today announced that the Food & Drug Administration granted orphan drug designation to LM-030, an investigational therapy licensed from Novartis and ready to enter into pivotal clinical trials for the treatment of Netherton Syndrome. “We are very pleased to have received the orphan drug designation for LM-030 which has demonstrated safety and clinical efficacy in a Phase I/II study. Netherton Syndrome is a severe genetic disorder with neonate onset that can be life-threatening. Receiving the orphan drug designation represents a significant step in the development of LM-030 for this devastating disease,” said Larry Hsu, LifeMax’s Co-founder and CEO, an industry veteran who previously founded and built Impax Laboratories into a publicly traded multi-billion dollar company. Orphan drug designation is granted by the FDA Office of Orphan Products Development to promote the development of investigational therapies for diseases affecting less than 200,000 people in the US. Benefits that come with an orphan drug designation include, but not limited to, tax credits for clinical development cost and seven-year market exclusivity. Netherton Syndrome is a severe autosomal recessive disease characterized by congenital erythroderma, “bamboo hair” and abnormality in the immune system. It can be life-threatening in pediatrics due to impaired skin barrier that leads to severe dehydration, hypernatremia, hypothermia, gross weight loss and sepsis. Failure to thrive is common in childhood as a result of chronic erythroderma, persistent cutaneous infection, malnutrition and metabolic disorders. The severity of the skin abnormality in older patients can fluctuate over time. Most Netherton Syndrome patients are also inflicted with immune system-related disorders such as food allergies and asthma. Though the exact prevalence of Netherton Syndrome is not well documented, it has a reported prevalence of 1-9 per 1,000,000.
LifeMax Laboratories , Inc .是一家专注于治疗罕见疾病且几乎没有治疗选择的私人公司,今天宣布食品和药物管理局授予 LM-030孤儿药称号。一种由诺华(Novartis)公司授权的研究疗法,准备进入治疗尼日顿综合征的关键临床试验。 “我们很高兴收到 LM-030的孤儿药名称,该名称在 I / II 期研究中证明了安全性和临床有效性。Netherton 综合征是一种严重的遗传性疾病,新生儿发病可能危及生命。LifeMax 的联合创始人兼首席执行官 LarryHsu 表示:“获得孤儿药物的指定标志着 LM-030在这一破坏性疾病的开发方面迈出了重要一步。”他是一位行业资深人士,曾创立 Impax 实验室,并将其打造成一家市值数十亿美元的上市公司。 孤儿药物的指定是由 FDA 孤儿产品开发办公室授予的,目的是促进开发研究治疗疾病的方法,在美国影响不到20万人。孤儿药物指定带来的好处包括但不限于临床开发成本的税收抵免和七年的市场独占性。 Netherton 综合征是一种严重的常染色体隐性疾病,以先天性红皮病、竹毛和免疫系统异常为特征。它可能危及生命的儿科由于受损的皮肤屏障,导致严重脱水,高钠血症,低体温,总体重减轻和败血症。由于长期的红皮病、持续的皮肤感染、营养不良和代谢紊乱,在儿童时期发育不良是常见的。老年患者皮肤异常的严重程度可能随时间而波动。大多数荷兰顿氏综合症患者也会出现与免疫系统相关的疾病,如食物过敏和哮喘。 虽然荷兰顿综合症的确切流行情况没有很好的文献记载,但据报道其患病率为每100万人1-9人。

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