Ravicti获准用于治疗尿素循环障碍

圣路易斯(MD Consult)——2013年2月1日，美国食品药品管理局(FDA)和Hyperion Therapeutics公司宣布，Ravicti (苯丁酸甘油酯)已获准用于部分2岁或以上尿素循环障碍(UCD)患者的长期治疗。Ravicti是一种氮结合剂。

氮是蛋白质代谢所产生的废弃产物，尿素循环可将其从血液中清除并转化为尿素，后者通过尿液排出体外。UCD患者体内氮蓄积，并以氨的形式存留在体内。

据Hyperion公司和FDA称，UCD属于遗传性代谢疾病，患者全身氨水平较高，而氨是一种潜在神经毒素。在UCD患者一生中，一旦氨水平升高，患者可反复出现高氨血症危象。并发症包括恶心、呕吐、头痛、昏迷和死亡。

Ravicti为液体制剂，每日3次，随餐服用，用于不能单纯通过蛋白限制饮食或氨基酸补充剂控制的UCD患者。Ravicti必须与蛋白限制饮食以及在某些情况下与膳食补充剂同时应用。

FDA批准Ravicti是基于10项临床试验数据，其中包括6项纳入UCD患者的研究。一项支持Ravicti安全性和有效性的主要研究纳入了44例服用Buphenyl(另一获批用于UCD控制的药物)的成人患者。患者随机服用Buphenyl或Ravicti 2周后交叉转换用药，再治疗2周。血液检查显示，Ravicti在控制氨水平方面与Buphenyl效果相当。另外针对儿童和成人的3项研究证实了Ravicti用于≥2岁患者的长期安全性和有效性。

Ravicti治疗患者最常见不良反应包括腹泻、腹胀和头痛。

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ST LOUIS (MD Consult) - On February 1, 2013, the US Food and Drug Administration (FDA) and Hyperion Therapeutics announced the approval of Ravicti (glycerol phenylbutyrate) for the chronic management of some urea cycle disorders (UCDs) in patients aged 2 years and older. Ravicti is a nitrogen-binding agent.

When protein is metabolized, it produces nitrogen as a waste product. The urea cycle removes nitrogen from the blood and converts it to urea, which is excreted in the urine. In persons with UCDs, nitrogen accumulates and remains in the body as ammonia.

According to Hyperion and the FDA, UCDs are a collection of inherited metabolic disorders in which affected patients experience high levels of systemic ammonia, a potent neurotoxin. Throughout their lives, patients with UCDs may experience recurrent hyperammonemic crises in which ammonia levels rise. Complications may include nausea, vomiting, headache, coma, and death.

Ravicti is a liquid and is taken 3 times a day with meals. It is intended for use in patients whose UCD cannot be managed by a protein-restricted diet or amino acid supplements alone. Ravicti must be used with a protein-restricted diet and, in some cases, dietary supplements.

The FDA approval of Ravicti was granted on the basis of data from 10 clinical trials, including 6 studies that included patients with UCD. The major study supporting Ravicti's safety and effectiveness involved 44 adults who had been receiving Buphenyl, another drug approved to control UCDs. Patients were randomly assigned to take Buphenyl or Ravicti for 2 weeks before being switched to the other product for an additional 2 weeks. Blood testing showed Ravicti was as effective as Buphenyl in controlling ammonia levels. Three additional studies in children and adults provided evidence supporting the long-term safety and effectiveness of Ravicti in patients aged 2 years and older.

The most common adverse effects in patients treated with Ravicti included diarrhea, flatulence, and headache.

Ravicti approved for the treatment of patients with urea cycle disorders