Approval is for primary hyperoxaluria type 1, which causes recurrent kidney stones and loss of kidney function
批准用于原发性高草酸尿1型，可导致复发性肾结石和肾功能丧失
Today, the U.S. Food and Drug Administration approved Oxlumo (lumasiran) as the first treatment for primary hyperoxaluria type 1 (PH1), a rare genetic disorder. This approval is a cumulation of the work of experts and community members coordinated by the Oxalosis & Hyperoxaluria Foundation and the Kidney Health Initiative.
今天，美国食品和药物管理局（U.S.Food and Drug Administration）批准Oxlumo（lumasiran）作为第一种治疗原发性高草酸尿1型（PH1）的药物，PH1是一种罕见的遗传性疾病。这项批准是专家和社区成员在草酸症和高草酸尿基金会和肾脏健康倡议的协调下所做的工作的累积。
“The approval of Oxlumo represents a great triumph of community involvement to address a rare disease. It is a result of input from patients, treating physicians, experts and sponsors at a patient-focused drug development meeting and through other collaborative efforts,” said Norman Stockbridge, M.D., Ph.D., director of the Division of Cardiology and Nephrology in the FDA’s Center for Drug Evaluation and Research.
Oxlumo的批准代表着社区参与解决一种罕见疾病的巨大胜利。FDA药物评估和研究中心心脏病和肾脏科主任诺曼·斯托克布里奇博士说：“这是患者、治疗医生、专家和赞助人在以患者为中心的药物开发会议上以及通过其他合作努力提供的结果。
Primary hyperoxalurias (PHs) are caused by excess production of oxalate, a substance consumed in food and also produced by the body. PH1 is the most common and severe type. PH1 affects an estimated one to three individuals per million in North America and Europe and accounts for approximately 80% of PH cases.
原发性高草酸尿（PHs）是由过量的草酸盐引起的，草酸盐是一种食物中消耗的物质，也是人体产生的物质。PH1是最常见和最严重的类型。据估计，在北美和欧洲，每百万人中有1到3人感染PH1，约占PH病例的80%。
Patients with PH1 produce far too much oxalate, which can combine with calcium to cause kidney stones and deposits in the kidneys. Patients can experience progressive kidney damage, which can lead to kidney failure and the need for dialysis (a treatment that purifies the blood). As kidney function worsens, oxalate can build up and damage other organs, including the heart, bones and eyes.
PH1患者产生过多的草酸，草酸与钙结合，导致肾结石和肾脏沉积。对于肾功能衰竭的患者来说，需要进行性的血液透析治疗。当肾功能恶化时，草酸会积聚并损害其他器官，包括心脏、骨骼和眼睛。
Oxlumo works to decrease oxalate production. It was evaluated in two studies in patients with PH1: a randomized, placebo-controlled trial in patients six years and older and an open-label study in patients younger than six years. Patients ranged in age from four months to 61 years at the first dose. In the first study, 26 patients received a monthly injection of Oxlumo followed by a maintenance dose every three months; 13 patients received placebo injections. The primary endpoint was the amount of oxalate measured in the urine over 24 hours. In the Oxlumo group, patients had, on average, a 65% reduction of oxalate in the urine, compared to an average 12% reduction in the placebo group. By the sixth month of the study, 52% of patients treated with Oxlumo reached a normal 24-hour urinary oxalate level; no patients treated with the placebo did.
Oxlumo可以降低草酸的产量。在PH1患者的两项研究中对其进行了评估：一项针对6岁及以上患者的随机安慰剂对照试验和一项针对6岁以下患者的开放标签研究。第一次给药的患者年龄从4个月到61岁不等。在第一项研究中，26名患者每月注射奥沙莫司，然后每三个月注射一次维持剂量；13名患者接受安慰剂注射。主要终点是24小时内尿液中草酸含量的测定。在 Oxlumo组，患者尿液中草酸的平均减少了65%，而安慰剂组的平均减少了12%。到研究的第六个月，52%的 Oxlumo治疗的患者达到24小时尿草酸水平；安慰剂治疗的患者没有达到。
In the second study, 16 patients younger than six years all received Oxlumo. Using another measure of oxalate in the urine, the study showed, on average, a 71% decrease in urinary oxalate by the sixth month of the study.
在第二项研究中，16名年龄小于6岁的患者都接受了 Oxlumo.莫治疗。采用另一种尿液中草酸的测量方法，研究表明，在研究的第六个月，尿草酸平均减少了71%。
The most common side effects of Oxlumo include injection site reaction and abdominal pain.
Oxlumo最常见的副作用包括注射部位反应和腹痛。
Oxlumo received orphan drug designation, which provides incentives to assist and encourage drug development for rare diseases. The application was also granted breakthrough therapy designation. In addition, the manufacturer received a rare pediatric disease priority review voucher. The FDA’s rare pediatric disease priority review voucher program is intended to encourage development of new drugs and biologics to prevent and treat rare diseases in children.
Oxlumo获得孤儿药称号，这为帮助和鼓励罕见疾病的药物开发提供了激励。该申请也获得了突破性的治疗指定。此外，厂家还收到了一张罕见的儿科疾病优先复查凭单。美国食品和药物管理局的罕见儿科疾病优先审查券计划旨在鼓励开发预防和治疗儿童罕见疾病的新药和生物制剂。
The FDA granted the approval of Oxlumo to Alnylam Pharmaceuticals, Inc.
FDA批准了Oxlumo给Alnylam制药公司。
德斯特DST • 服务项目
认证咨询（FDA/WHO/中国GMP）
计算机化系统验证丨工艺核对咨询
实验室管理丨药厂厂房设计
模拟GMP认证检查丨数据可靠性审计
GMP合规性审计丨药厂管理咨询
厂房GMP认证丨医疗器械GMP认证