FDA首次批准兰伯特·伊顿肌无力综合征新药一一amifampridine

日期:2019-01-15    作者:林佑       分享 :

美国食品药物管理局(FDA)近月批准一种名为amifampridine的片剂,这是用治罕见疾病一一成人兰伯特·伊顿肌无力综合征新药。



兰伯特-伊顿肌无力综合征(英文名:Lambert-Eaton myasthenic syndrome,简写:LEMS)是一种罕见的免疫性疾病。具体症状为全身肌肉无力丶疲累乏力,长此下去,因抵抗力下降引起众多并发症。



FDA药物评价研究中心专家兼神经学产品部主任Billy Dunn医学博士提到,兰伯特-伊顿肌无力综合征是一类比较罕见的慢性疾病,约每百万人就有三至十人潜在患者,他/她们需要长期服用药物来控制病情,否则会容易感到疲倦,这无疑对日常生活造成困扰。



在病理学研究上,研究员发现LEMS会干扰自身免疫析别功能,令其攻击身体的神经网络,阻碍神经元与肌肉之间的连系(即是阻断神经细胞向肌肉细胞发送信号),更甚的是,LEMS会引发其他的免疫性疾病,例如1型糖尿病,格雷夫斯病,多发性硬化症,类风湿性关节炎等等,甚至会提高癌症罹患风险(常见小细胞肺癌)。



临床试验针对amifampridine的疗效进行了研究。该项实验招募了64名确诊LEMS的成年患者,随机分为病理组和对照组,前者服用amifampridine,后者服用常规药物。评估方法包括13项医生对重症肌无力患者的定量评分、7项健康状态的总体印象评分。评估结果均显示amifampridine药物相对常规药物疗效更佳。



在临床毒理试验中显示,amifampridine没有明显副作用,只有部分患者会出现头痛、背痛、腹痛、恶心、肌肉痉挛等症状。体验发现呈现肝酶与血压上升趋势。



此外,亦有可能引起肠胃不适症状(烧灼或刺痛异常)、伤风感冒症状(上呼吸道感染)、皮肤过敏(皮疹、荨麻疹、皮肤瘙痒)、引发癫痫。如有服用异常迹象应立即救医。





FDA approves first treatment for Lambert-Eaton myasthenic syndrome, a rare autoimmune disorder



November 28, 2018


The U.S. Food and Drug Administration today approved Firdapse (amifampridine) tablets for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in adults. LEMS is a rare autoimmune disorder that affects the connection between nerves and muscles and causes weakness and other symptoms in affected patients. This is the first FDA approval of a treatment for LEMS.



“There has been a long-standing need for a treatment for this rare disorder,” said Billy Dunn, M.D., director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research. “Patients with LEMS have significant weakness and fatigue that can often cause great difficulties with daily activities.”



In people with LEMS, the body’s own immune system attacks the neuromuscular junction (the connection between nerves and muscles) and disrupts the ability of nerve cells to send signals to muscle cells. LEMS may be associated with other autoimmune diseases, but more commonly occurs in patients with cancer such as small cell lung cancer, where its onset precedes or coincides with the diagnosis of cancer. The prevalence of LEMS is estimated to be three per million individuals worldwide.



The efficacy of Firdapse was studied in two clinical trials that together included 64 adult patients who received Firdapse or placebo. The studies measured the Quantitative Myasthenia Gravis score (a 13-item physician-rated categorical scale assessing muscle weakness) and the Subject Global Impression (a seven-point scale on which patients rated their overall impression of the effects of the study treatment on their physical well-being). For both measures, the patients receiving Firdapse experienced a greater benefit than those on placebo.



The most common side effects experienced by patients in the clinical trials were burning or prickling sensation (paresthesia), upper respiratory tract infection, abdominal pain, nausea, diarrhea, headache, elevated liver enzymes, back pain, hypertension and muscle spasms. Seizures have been observed in patients without a history of seizures. Patients should inform their health care provider immediately if they have signs of hypersensitivity reactions such as rash, hives, itching, fever, swelling or trouble breathing.



The FDA granted this application Priority Review and Breakthrough Therapy designations. Firdapse also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

 

The FDA granted the approval of Firdapse to Catalyst Pharmaceuticals, Inc.



The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.




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