简介:
部份中文西罗莫司处方资料(仅供参考)
商品名:Rapalimus Gel 0.2%
英文名:Sirolimus
中文名:西罗莫司外用凝胶
生产商:诺贝尔制药公司
药品简介
2018年3月23日,Nobelpharma Co.Ltd宣布,孤儿药Rapalimus(Sirolimus,西罗莫司)凝胶0.2%在日本获批上市,用于治疗结节性硬化综合征中的血管纤维瘤。
结节性硬化症是一种罕见的遗传疾病,可导致发育障碍和肿瘤病变覆盖整个身体,如大脑、皮肤和肾脏。其中,皮肤病变如血管纤维瘤经常发生,症状在青春期变得明显,给病人降低他们的生活质量。然而,目前的治疗方法是侵入性的,例如手术或激光治疗难以用于幼儿或发育障碍患者。
ラパリムスゲル0.2%
药物分类名称
与结节性硬化症相关的皮肤病变治疗剂(mTOR抑制剂)
批准日期:2018年6月
商標名
Rapalimus Gel 0.2%
日本名:シロリムス
英 名:Sirolimus
化学名:(1R, 9S, 12S, 15R, 16E, 18R, 19R, 21R, 23S, 24E, 26E, 28E, 30S, 32S, 35R)-1, 18-ジヒドロキシ-12-{(1R) -2-[(1S, 3R, 4R)-4-ヒドロキシ-3-メトキシシクロヘキシル]-1-メチルエチル}-19, 30-ジメトキシ-15, 17, 21, 23, 29, 35-ヘキサメチル-11, 36-ジオキサ-4-アザトリシクロ[30. 3. 1. 04, 9]ヘキサトリアコンタ-16, 24, 26, 28-テトラエン-2, 3, 10, 14, 20-ペンタオン
分子式:C51H79NO13
分子量:914.17
構造式:
性 状:
白色至灰白色粉末
熔点:180至185℃(分解)
批准条件
1.制定药品风险管理计划并适当实施。
2.由于国内的临床试验案例极为有限,通过对所有案例进行使用结果调查,直到制造和销售后累计一定数量的案例数据为止, 除了掌握使用该药物的患者的背景信息外,还应尽快收集有关该药物安全性和有效性的数据,并采取必要措施正确使用该药物。
药用药理学
据报道,当在裸鼠的结节硬化模型中经皮给予西罗莫司(局部应用软膏)时,西罗莫司显示出肿瘤生长抑制作用。
适应症
与结节性硬化症相关的皮肤损伤
用法与用量
每天两次将适量的量涂抹在患处。
包装
10克/管
制造供应商
June 6, 2018, the mTOR inhibitor sirolimus topical gel formulation (trade name Laparimus gere 0.2%) was released. Adaptation is "skin lesion associated with tuberous sclerosis", dosage regimen is "twice a day, apply an appropriate amount to the affected area". As an identical ingredient, an oral preparation (tablet) is clinically used from December 2014 as an indication for "lymphatic vascular myoma".
Tuberous sclerosis (TSC) is a rare disease of autosomal dominant inheritance characterized by systemic hamartoma (a lesion of intermediate character between tumor and malformation). As the causative gene of TSC, TSC1 gene on chromosome 9 or TSC2 gene on chromosome 16 has been identified, and due to abnormality of these genes downstream mTOR (mammalian rapamycin target protein) is activated and cell proliferation Etc. are promoted. As a result, cell growth and proliferation are enhanced, apoptosis is suppressed, benign tumors are generated in almost the whole body such as skin, brain, lung, heart, kidney and bone, and central nervous symptoms such as behavioral abnormalities, mental development delay, epilepsy .
Everolimus oral formulation (affinitol), an mTOR inhibitor, has been clinically used for various diseases caused by TSC. However, the indication for everolimus is renal angiomyolipomatome and subcapillary giant cell astrocytoma associated with TSC, and for skin lesions of facial angiofibroma and fibrous head aspect (or forehead fibrous aspect) There were only lasers, freezing coagulation using liquid nitrogen, or surgical treatment. However, these are highly invasive and many problems remain such as postoperative scarring.
The sirolimus topical gel formulation is the first drug in the world that can be expected to have a local effect noninvasively as a therapeutic drug for TSC skin lesion. In a 12-week twice-daily placebo-controlled double-blind study (domestic verification test) for Japanese TSC patients, efficacy in angiofibroma, etc. and safety in long-term study (12 months) The sex was also confirmed. This formulation was designated as a target item of the preliminary examination designation system in October 2015 and was designated as a drug for rare diseases in December of the same year.
Adverse reactions are recognized in 70.3% from clinical trial until approval. The main side effects were skin dryness (30.4%), application site irritation feel (27.0%), acne (10.1%), pruritus (8.8%), acne-like dermatitis (6.1%), eye irritation (5.4% )Such.
Since photosensitivity may develop, it is necessary to avoid excessive ultraviolet exposure by sunlight or sunburn lamps when using the medicine, or if improvement of the symptoms is not observed within 12 weeks of treatment start, Review the necessity, keep in mind that you do not continue to take medication as soon as possible.
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