Procysbi获准用于治疗肾病型胱氨酸症

圣路易斯(MD Consult)——2013年4月30日，美国食品药品管理局(FDA)和Raptor Pharmaceuticals宣布，Procysbi(半胱胺重酒石酸氢盐)缓释胶囊已获准用于治疗成年人和≥6岁儿童的肾病型胱氨酸症。胱氨酸耗竭是肾病型胱氨酸症的主要治疗策略。

据Raptor介绍，肾病型胱氨酸症占胱氨酸症的95%。胱氨酸症是一种罕见、危及生命的代谢性溶酶体储存障碍，可导致毒性胱氨酸在所有细胞、组织和器官中累积。胱氨酸水平升高可引起进行性、不可逆的组织损伤和多器官衰竭，包括肾衰、失明、肌肉萎缩和过早死亡。肾病型胱氨酸症通常发生在婴儿期，并且需要终身治疗。据估计全美国目前有大约500例胱氨酸症患者健在。

FDA批准Procysbi是基于6项临床试验的数据，包括1项纳入43例肾病型胱氨酸症患者的多中心、随机、活性对照的3期试验。在这项3期试验中，接受Procysbi治疗的患者在整个12 h剂量期中始终保持胱氨酸耗竭。这些患者此前从未能维持住胱氨酸水平。既往多项研究显示，持续胱氨酸耗竭可明显推迟疾病进展，包括肾功能不全、透析、肾移植、器官衰竭和过早死亡。

在研究中最常报告的不良反应为呕吐、腹痛、不适、头痛、恶心、腹泻、厌食/食欲下降、口臭、疲乏、头晕、皮肤异味和皮疹。Procysbi禁用于对青霉胺过敏的患者。半胱氨的使用与可逆性白细胞减少和肝功能异常有关。因此，应监测血细胞计数和肝功能。

目前，针对年龄＜6岁的儿童的临床研究仍在进行中，以评估Procysbi对此类患者的安全性和有效性。

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ST LOUIS (MD Consult) - On April 30, 2013, the US Food and Drug Administration (FDA) and Raptor Pharmaceuticals announced the approval of Procysbi (cysteamine bitartrate) delayed-release capsules for the management of nephropathic cystinosis in adults and children aged 6 years and older. Cystine depletion is the primary treatment strategy for nephropathic cystinosis.

According to Raptor, nephropathic cystinosis comprises 95% of cystinosis, a rare, life-threatening metabolic lysosomal storage disorder that causes toxic accumulation of cystine in all cells, tissues, and organs in the body. Elevated cystine levels lead to progressive, irreversible tissue damage and multi-organ failure, including kidney failure, blindness, muscle wasting, and premature death. Nephropathic cystinosis is usually diagnosed in infancy and requires lifelong therapy. Left untreated, the disease is usually fatal by the end of the first decade of life. An estimated 500 patients with cystinosis live in the United States.

The FDA approval of Procysbi was granted on the basis of data from 6 clinical trials, including a multi-center, randomized, active-controlled, phase 3 trial of 43 patients with nephropathic cystinosis and extension data from that trial. In a phase 3 study, patients who received Procysbi showed consistent cystine depletion over a full 12-hour dosing period. Sustained levels of cysteamine have not historically been achieved in the majority of patients in this population. Studies have shown that sustained cystine depletion in patients may significantly delay disease progression, including kidney dysfunction, dialysis, kidney transplant, organ failure, and premature death.

The most commonly reported adverse reactions in studies were vomiting, abdominal pain, discomfort, headaches, nausea, diarrhea, anorexia/decreased appetite, breath odor, fatigue, dizziness, skin odor, and rash.

Procysbi is contraindicated in patients with hypersensitivity to penicillamine. The use of cysteamine has been associated with reversible leukopenia and abnormal liver function studies. Therefore, blood counts and liver function tests should be monitored.

Clinical studies are ongoing in children younger than 6 years to evaluate the safety and efficacy of Procysbi.

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