利维肤治疗药公司宣布FDA已接受布西拉明(bucillamine)的研究性新药申请,在美国进行治疗胱氨酸尿症Ⅱ期临床试验。FDA已认定该药为治疗胱氨酸尿症罕见病药。胱氨酸尿症是常染色体隐性遗传性疾病,患者尿中出现大量胱氨酸,导致肾结石。此种肾结石块较大,常多发,可致肾功能丧失。全球发病率为 Levi Skin Therapeutics, Inc. announced that it has received an investigational new drug application from bucillamine to conduct Phase II clinical trials of cystinemia in the United States. FDA has identified the drug as a rare cure for cystineuria. Cystineuria is an autosomal recessive disease, a large number of patients with cystine in urine, leading to kidney stones. Such large kidney stones, often multiple, can cause loss of renal function. The global incidence rate is