On June 6, 2022, the FDA expanded the indications for mycophenolate mofetil (MMF) to include the prophylaxis of organ rejection in combination with other immunosuppressants in pediatric recipients of allogeneic heart or liver transplants aged 3 months and older. The approved oral dosing regimen for these patients was a starting dose of 600 mg/m2 with titration up to a maximum of 900 mg/m2 twice daily. Data to support efficacy in pediatric patients were derived from established pharmacokinetic (PK) relationships across approved populations, a PK study in pediatric liver transplant recipients, and information from the Scientific Registry of Transplant Recipients database. Information supporting safety was based on comparing mycophenolic acid (MPA) exposure with that in pediatric kidney transplant recipients, the published literature, and post‐marketing safety reports. Efficacy in pediatric patients was established based on extrapolation of efficacy from studies in adult liver, adult heart, and pediatric kidney transplant populations, and similarity in MPA exposure between pediatric and adult patients. Review of the data supported an oral dosing regimen for pediatric heart transplant and liver transplant recipients consisting of a starting dose of 600 mg/m2 up to a maximum of 900 mg/m2 b.i.d. A dosage range for MMF is recommended recognizing that the MMF dose may be modified in clinical practice for myriad factors. The dosage recommendations in the labeling for pediatric liver and pediatric heart transplant patients are intended to permit individualized dosing based on clinical assessment of these factors.

中文翻译：

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批准霉酚酸酯用于预防儿童心脏或肝脏移植受者的器官排斥：监管角度

2022 年 6 月 6 日，FDA 扩大了吗替麦考酚酯 (MMF) 的适应症，包括与其他免疫抑制剂联合用于 3 个月及以上接受同种异体心脏或肝脏移植的儿科患者预防器官排斥。这些患者的批准口服给药方案的起始剂量为 600 mg/m22滴定浓度最高可达 900 mg/m2每天两次。支持儿科患者疗效的数据来自已批准人群中已建立的药代动力学 (PK) 关系、儿科肝移植受者的 PK 研究以及移植受者科学登记数据库的信息。支持安全性的信息基于霉酚酸 (MPA) 暴露与儿童肾移植受者的暴露比较、已发表的文献和上市后安全报告。儿科患者的疗效是根据成人肝脏、成人心脏和儿科肾移植人群研究的疗效外推，以及儿科和成人患者 MPA 暴露的相似性而确定的。数据审查支持儿童心脏移植和肝移植受者的口服给药方案，起始剂量为 600 mg/m22最高可达 900 mg/m2bid 建议MMF的剂量范围，因为认识到MMF剂量可能在临床实践中因多种因素而修改。儿童肝脏和儿童心脏移植患者标签中的剂量建议旨在允许根据这些因素的临床评估进行个体化剂量。