总览

Myalepta是一种除饮食外还用于治疗脂肪营养不良的药物，其中患者的皮肤下脂肪组织丢失，体内其他部位（例如肝脏和肌肉）的脂肪堆积。该药物用于：

2岁以上的成年人和儿童，全身（全身）脂肪营养不良（贝拉迪内利-塞普综合征和劳伦斯综合征）；

标准治疗失败时，患有部分（局部）脂肪营养不良（包括Barraquer-Simons综合征）的12岁以上成人和儿童。

Myalepta包含活性物质metreleptin。

由于患有各种形式的脂肪营养不良的患者数量很少，这些疾病被认为是“罕见的”，并且Myalepta在2012年7月17日被指定为“孤儿药”（一种罕见疾病中使用的药物）。可以在欧洲药品管理局的网站上找到：ema.europa.eu/查找医学/人类药物/罕见病（Bernardinelli-Seip综合征：12/7/12； Lawrence综合征：12/7/12；家族性部分脂肪营养不良： （2012年7月17日；巴拉克·西蒙斯综合征（Barraquer-Simons syndrome）：12年7月17日）。

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如何使用Myalepta？

Myalepta仅可通过处方获得，治疗应由在代谢紊乱患者的诊断和管理方面经验丰富的医生开始和监测。

每天在同一时间在腹部（腹部），大腿或上臂的皮肤下每天注射Myalepta。建议的每日剂量取决于患者的体重，并根据患者对治疗的反应进行调整。患者或护理人员一旦接受培训，便可以自己注射药物。

有关使用Myalepta的更多信息，请参阅包装传单或与您的医生或药剂师联系。

Myalepta如何运作？

脂肪营养不良的患者体内的人体激素-瘦素水平低，它在调节体内脂肪和糖的分解中起关键作用。这会导致皮肤下脂肪的流失以及在肝脏和肌肉等部位的脂肪积聚，以及血液中高水平的脂肪。它还会导致胰岛素抵抗（当人体无法识别胰岛素时，一种有助于调节血糖水平的激素）。

Myalepta中的活性物质metreleptin与leptin相似。它替代瘦素并增加血液，肌肉和肝脏中的脂肪分解，从而纠正脂肪营养不良患者的某些异常，包括胰岛素抵抗。但是，这种药物不能恢复皮肤下的脂肪组织。

研究显示Myalepta有哪些好处？

Myalepta在两项涉及107名患有一般性或部分性脂肪营养不良的成人和儿童的主要研究中已显示出可有效降低血脂水平。在研究中，未将Myalepta与其他任何治疗方法进行比较。经过12个月的治疗，泛发性疾病患者的血脂（甘油三酸酯）水平从约15 mmol / l降至约5 mmol / l，部分疾病患者的血脂水平从约16 mmol / l降至约6 mmol / l 。

胰岛素抵抗也得到了改善：一种称为糖基化血红蛋白（HbA1c）的血液水平从8.6％降至6.4％，从8.8％降至8.0，从8.6％降至6.4％，这表明血糖的控制水平。部分脂肪营养不良患者的百分比。

Myalepta有哪些风险？

Myalepta最常见的副作用（可能影响超过十分之一的人）是低血糖症（低血糖）和体重减轻。有关Myalepta副作用和限制的完整列表，请参阅包装手册。

为什么Myalepta在欧盟获得授权？

业已证明，Myalepta可纠正脂肪营养不良患者的瘦素缺乏症所引起的某些异常，这是一种罕见的疾病，很少有治疗方法。 Myalepta所见的副作用属于此类治疗所预期的那种。因此，欧洲药品管理局认为Myalepta的好处大于其风险，因此可以授权在欧盟使用。

Myalepta已在“特殊情况”下获得授权。这是因为由于这种病的罕见性，所以无法获得有关Myalepta的完整信息。原子能机构每年将审查可获得的任何新信息，并将根据需要更新此概述。

Myalepta还在等待什么信息？

由于Myalepta已在特殊情况下获得授权，因此销售Myalepta的公司将建立一个治疗药物患者的注册中心，并进行研究以进一步调查治疗的益处和风险，包括Myalepta触发抗体产生的可能性。

正在采取什么措施来确保安全有效地使用Myalepta？

销售Myalepta的公司将向患者和医生提供教育材料，其中包括有关如何使用该药物以及在发生包括过敏反应，低血糖症和严重感染在内的副作用的情况下该怎么办的详细信息。

产品特性和包装手册的摘要中还包括医疗专业人员和患者应遵循的有关安全有效使用Myalepta的建议和预防措施。

对于所有药物，均不断监测Myalepta的使用数据。仔细评估了Myalepta报告的副作用，并采取了任何必要的措施来保护患者。

Overview

Myalepta is a medicine used in addition to diet to treat lipodystrophy, where patients have loss of fatty tissue under the skin and build-up of fat elsewhere in the body such as in the liver and muscles. The medicine is used in:

· adults and children above the age of 2 years with generalised (throughout the body) lipodystrophy (Berardinelli-Seip syndrome and Lawrence syndrome);

· adults and children above the age of 12 years with partial (localised) lipodystrophy (including Barraquer-Simons syndrome), when standard treatments have failed.

Myalepta contains the active substance metreleptin.

Because the number of patients with the various forms of lipodystrophy is low, the diseases are considered ‘rare’, and Myalepta was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 17 July 2012. Further information on the orphan designations can be found on the European Medicines Agency’s website: ema.europa.eu/Find medicine/Human medicines/Rare disease designation (Bernardinelli-Seip syndrome: 17/07/12; Lawrence syndrome: 17/07/12; familial partial lipodystrophy: 17/07/12; Barraquer-Simons syndrome: 17/07/12).

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How is Myalepta used?

Myalepta can only be obtained with a prescription and treatment should be started and monitored by a doctor experienced in the diagnosis and management of patients with metabolic disorders.

Myalepta is given as a daily injection under the skin of the abdomen (belly), thigh or upper arm, at the same time every day. The recommended daily dose depends on the patient’s bodyweight and is adjusted based on the patient’s response to treatment. Patients or carers can inject the medicine themselves once they have been trained.

For more information about using Myalepta, see the package leaflet or contact your doctor or pharmacist.

How does Myalepta work?

Patients with lipodystrophy have low levels of a human hormone called leptin, which plays a key role in regulating the break-down of fats and sugars in the body. This leads to loss of fat from under the skin and its build-up in places such as the liver and muscles, as well as high levels of fat in the blood. It also results in insulin resistance (when the body is unable to recognise insulin, a hormone that helps regulate blood sugar levels).

The active substance in Myalepta, metreleptin, is similar to leptin. It replaces leptin and increases fat breakdown in the blood, muscles and liver, thereby correcting some abnormalities in patients with lipodystrophy, including insulin resistance. However, the medicine does not restore fat tissue under the skin.

What benefits of Myalepta have been shown in studies?

Myalepta has been shown to be effective at lowering blood fat levels in 2 main studies involving a total of 107 adults and children with generalised or partial lipodystrophy. In the studies Myalepta was not compared with any other treatment. After 12 months of treatment, blood levels of fats (triglycerides) decreased from around 15 mmol/l to around 5 mmol/l in patients with generalised disease, and from around 16 mmol/l to around 6 mmol/l in patients with partial disease.

Insulin resistance was also improved: blood levels of a substance called glycosylated haemoglobin (HbA1c), which gives an indication of how well the blood glucose is controlled, fell from 8.6% to 6.4% in patients with generalised lipodystrophy, and from 8.8% to 8.0% in patients with partial lipodystrophy.

What are the risks associated with Myalepta?

The most common side effects with Myalepta (which may affect more than 1 in 10 people) are hypoglycaemia (low blood glucose) and weight loss. For the full list of side effects and restrictions with Myalepta, see the package leaflet.

Why is Myalepta authorised in the EU?

Myalepta has been shown to correct some abnormalities caused by leptin deficiency in patients with lipodystrophy, a rare condition for which few treatments are available. Side effects seen with Myalepta are of the kind expected from this type of treatment. The European Medicines Agency therefore decided that Myalepta’s benefits are greater than its risks and it can be authorised for use in the EU.

Myalepta has been authorised under ‘exceptional circumstances’. This is because it has not been possible to obtain complete information about Myalepta due to the rarity of the condition. Every year, the Agency will review any new information that becomes available and this overview will be updated as necessary.

What information is still awaited for Myalepta?

Since Myalepta has been authorised under exceptional circumstances, the company that markets Myalepta will set-up a registry of patients treated the medicine, and conduct studies to further investigate the benefits and risks of treatment including the possibility for Myalepta to trigger the production of antibodies.

What measures are being taken to ensure the safe and effective use of Myalepta?

The company that markets Myalepta will provide educational materials to patients and doctors with detailed information on how to use the medicine and what to do in case of side effects, including allergic reactions, hypoglycaemia and serious infections.

Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Myalepta have also been included in the summary of product characteristics and the package leaflet.

As for all medicines, data on the use of Myalepta are continuously monitored. Side effects reported with Myalepta are carefully evaluated and any necessary action taken to protect patients.

Other information about Myalepta

Myalepta received a marketing authorisation valid throughout the EU on 30 July 2018.