Cystaren获准用于治疗胱氨酸病患者的角膜胱氨酸结晶

圣路易斯(MD Consult)——10月4日，Sigma-Tau Pharmaceuticals公司宣布美国食品药品管理局(FDA)已批准Cystaran(半胱胺滴眼液)0.44%用于治疗胱氨酸病患者的角膜胱氨酸结晶。半胱胺是一种胱氨酸耗竭剂，可降低胱氨酸病患者细胞中胱氨酸的含量。然而，口服给药时，半胱胺不能到达角膜，因此不能有效降低胱氨酸病对眼睛的影响。目前，Cystaran是唯一被FDA批准用于治疗此适应证的眼科药物。

胱氨酸病是一种罕见的遗传溶酶体蓄积病，以胱氨酸异常蓄积为特征。该病可导致胱胺酸结晶蓄积在机体的不同器官，包括角膜、肾脏、肝脏、胰腺、肌肉、大脑和白细胞。角膜胱氨酸蓄积可导致眼部并发症，如斜视、异物感、视力改变、角膜混浊和畏光。其他并发症还包括肌肉无力、糖尿病、甲状腺功能减退、吞咽困难和佝偻病。Sigma Tau的数据显示，美国约有300名儿童和年轻成人罹患胱氨酸病，全球的该病患者数量约为2,000例。

此前由国立卫生研究院开展的临床对照研究在约300例患者中对Cystaran的临床安全性和疗效进行了评估，结果表明该药可有效治疗角膜胱氨酸结晶。最常见的眼部不良反应为对光敏感、潮红、眼睛疼痛/刺激、头痛和视野缺损。

未来，Cystaran将通过专业药店分销渠道销售。

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ST LOUIS (MD Consult) - On October 4, 2012, Sigma-Tau Pharmaceuticals announced that the US Food and Drug Administration (FDA) has approved Cystaran (cysteamine ophthalmic solution) 0.44% for the treatment of corneal cystine crystals in patients with cystinosis. Cysteamine is a cystine-depleting agent that lowers the cystine content of cells in patients with cystinosis. However, when orally administered, cysteamine does not reach the cornea and is therefore ineffective in reducing the ocular effects of cystinosis. At this time, Cystaran represents the only FDA-approved ophthalmic treatment for this condition.

According to Sigma Tau, cystinosis affects approximately 300 children and young adults in the United States and 2,000 persons worldwide. Cystinosis is a rare, genetic lysosomal storage disease, characterized by the abnormal accumulation of the amino acid, cystine. The disease causes cystine crystals to accumulate in various organs of the body, including the corneas, kidneys, liver, pancreas, muscles, brain. and white blood cells. Corneal cystine accumulation can lead to ocular complications such as squinting, foreign body sensations, changes in visual acuity, corneal haziness, and photophobia. Other complications of cystinosis include muscle weakness, diabetes, hypothyroidism, difficulty swallowing, and rickets.

The clinical safety and efficacy of Cystaran were previously evaluated in controlled clinical trials conducted by the National Institutes of Health in approximately 300 patients. Results of these studies support the use of ophthalmic cysteamine as an effective treatment of corneal cystine crystals. The most frequently reported ocular adverse reactionswere sensitivity to light, redness, eye pain/irritation, headache, and visual field defects.

Cystaran is planned to be available in the future through specialty pharmacy distribution channels.

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