FDA批准肉毒中毒抗毒素

圣路易斯(MD Consult)——2013年3月22日与25日，美国食品药品管理局(FDA)和Cangene公司宣布，肉毒中毒抗毒素Heptavalent(马)已获准用于治疗可疑的或明确的肉毒杆菌神经毒素A、B、C、D、E、F或G暴露。这种产品是用马血浆制备的，所含的抗体片段混合物可中和已知能导致肉毒中毒的所有7种肉毒杆菌神经毒素血清型。它是迄今唯一种被美国批准用于治疗自然发生的非婴儿肉毒中毒和急诊患者的肉毒中毒抗毒素。

肉毒杆菌毒素是由肉毒杆菌分泌的一种神经毒素，可导致严重麻痹即肉毒中毒。自然条件下，肉毒中毒主要见于不恰当摄入加工食品，或在病菌暴露后发生定植的婴儿或成人。由于肉毒杆菌毒素具有很强的毒力和潜在致死性，美国将其列入了最高优先级生物恐怖主义威胁的物质名单。该产品将被成为美国战略储备的一部分，以备紧急状况响应所需。

该产品的有效性在动物中得到了检验，原因是开展人体有效性研究是不可行或不符合伦理的。动物研究的结果表明，这种抗毒素理论上对人类肉毒中毒患者是有益的。根据动物条例，FDA可在高质量动物研究结果提示理论上对人体有效，且已确认人类使用的安全性的情况下批准药物。这是首个依据动物条例获得批准的血浆衍生药物。

该产品的安全性已在40名健康人和228例经验性使用该抗毒素的患者中得到了检验。观察到的最常见不良反应包括头痛、发热、寒战、皮疹、瘙痒及恶心。由于该产品是用马血浆制备的，它有可能导致对马蛋白敏感的患者发生过敏反应和迟发型超敏反应。

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ST LOUIS (MD Consult) - On March 22 and 25, 2013, the US Food and Drug Administration (FDA) and Cangene announced the approval of Botulism Antitoxin Heptavalent (equine) for the treatment of suspected or documented exposure to the botulinum neurotoxin A, B, C, D, E, F or G. The product is derived from horse plasma and contains a mixture of antibody fragments that neutralize all 7 of the botulinum nerve toxin serotypes known to cause botulism. It is the only botulism antitoxin available in the United States for treating naturally occurring, non-infant botulism, and for administering to patients under emergency conditions.

Botulinum toxin is a nerve toxin produced by the bacterium Clostridium botulinum that causes a serious paralytic illness known as botulism. Naturally occurring cases are mainly seen in infants or in adults who have consumed improperly processed foods or have been colonized through exposure to the bacteria. Because of the the extreme potency and potential lethality of botulinum toxin, it has been identified in the United States as one of the highest priority bioterrorism threats. The product is to be stored in the US Strategic National Stockpile for emergency preparedness and responses.

The effectiveness of the product was studied in animals because it was not feasible or ethical to conduct efficacy studies in humans. These results provided substantial evidence that the antitoxin is reasonably likely to benefit humans with botulism. Under the FDA's Animal Rule, the agency may approve a biologic product when the results of well-controlled animal studies demonstrate that the product is reasonably likely to be effective, in addition to establishing safety in humans. This is the first approval of a plasma derivative using the Animal Rule.

The safety of the product was tested in 40 healthy persons and also monitored in 228 patients who received the antitoxin experimentally under a botulism treatment program administered by the Centers for Disease Control and Prevention. The most commonly observed adverse effects were headache, fever, chills, rash, itching, and nausea. Because the product is manufactured from horse plasma it may cause allergic reactions and a delayed hypersensitivity reaction (serum sickness) in persons sensitive to horse proteins.

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