2013年4月30日，美国食品与药物管理局（FDA）批准了Procysbi（半胱胺重酒石酸氢盐）用于儿童和成人肾病型胱胺酸症（ nephropathic cystinosis ）的治疗。Procysbi是一种缓释胶囊剂，用于年龄≥6岁的患者，每12小时使用一次。
半胱胺药物治疗患者常见的不良反应有恶心、口臭、腹痛、便秘、消化不良或反胃、头痛、嗜睡和头晕。其他少见但严重的副作用有胃或肠溃疡或出血、精神状态改变、癫痫、严重皮疹和过敏反应。
支持半胱胺重酒石酸氢盐安全性和有效性的主要研究为纳入43名患有肾病型胱胺酸症的成人及儿科患者的临床试验。试验中，患者在头三周时间内被随机配给Cystagon或Procysbi，然后再切换到其它药品治疗三周。血液检测结果显示Procysbi在控制胱胺酸水平上与Cystagon一样有效。
胱氨酸病是一种罕见遗传性疾病，估计在美国有500人，全球约3000人正受此疾病的困扰。这种疾病如果在儿童早期不进行治疗会有致命危险，胱氨酸病能引起胱氨酸在人体每个细胞中的聚集。胱氨酸的蓄积会导致一些肾脏问题，使人体内太多的糖，蛋白质和盐通过尿液排出。胱氨酸病可能会导致身体发育迟缓，使身材矮小，骨骼脆弱并增长缓慢，使肾功能衰竭恶化。胱氨酸病有三种类型，其中最为严重是肾病型胱胺酸病，这种病能严重损坏人体的肾脏。
目前，FDA批准用于治疗胱氨酸症的药物有Cystagon（半胱胺重酒石酸氢盐），是1994年批准的一种速释药片，需昼夜不停地每6小时服用一次以控制胱氨酸的水平；还有Cystaran滴眼剂（半胱胺溶液型滴眼剂），去年被批准用于治疗角膜胱氨酸晶体堆积
http://dailymed.nlm.nih.gov/dailymed/lookup.cfm?setid=b7b6e290-5168-46dc-9e7f-5995420ec1c1
PROCYSBI Rx
Generic Name and Formulations:
Cysteamine bitartrate 25mg, 75mg; delayed-release caps.

Company:
Raptor Pharmaceuticals, Inc.
Indications for PROCYSBI:
Management of nephropathic cystinosis.

Adults and Children's Dose:
<6yrs: not established. Take on an empty stomach. Swallow whole or sprinkle contents of cap on 4oz of food (eg, applesauce or berry jelly) or mix in 4oz of liquids (eg, orange or apple juice); also, may administer via feeding tube after mixing with 4oz of applesauce. ≥6yrs: New patients: start on ⅙ to ¼ of the maintenance dose; increase gradually over 4–6 weeks. Maintenance: 1.3g/m2/day, in 2 divided doses given every 12 hrs. Max 1.95g/m2/day. Goal of therapy: to maintain WBC cystine level <1nmol ½ cystine/mg protein or a plasma cysteamine concentration >0.1mg/L. Switching from immediate-release cysteamine: give previous total daily dose of immediate-release caps. Dose titration, measuring WBC cystine levels: see full labeling.

Pharmacological Class:
Cystine-depleting agent.

Contraindications:
Penicillamine hypersensitivity.

Warnings/Precautions:
Monitor for development of skin or bone lesions and interrupt dosing if occur; may restart at a lower dose. Discontinue if severe skin rash develops. Consider lowering the dose if severe GI symptoms develop. eva luate and monitor for CNS symptoms; interrupt or adjust dose if severe symptoms, persist or progress. Monitor blood counts and alkaline phosphatase levels. Monitor for signs/symptoms of benign intracranial hypertension. Pregnancy (Cat.C). Nursing mothers: not recommended.

Adverse Reactions:
Vomiting, abdominal pain/discomfort, headaches, nausea, diarrhea, anorexia/decreased appetite, breath odor, fatigue, dizziness, skin odor, rash; skin or bone lesions, leukopenia, CNS effects (eg, somnolence, encephalopathy, seizures), GI ulceration/bleeding, interstitial nephritis, elevated alkaline phosphatase, pseudotumor cerebri, papilledema.

How Supplied:
Caps 25mg—60; 75mg—250