Travere Therapeutics, Inc. has announced that the US Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA) for FILSPARI® (sparsentan) for the treatment of focal segmental glomerulosclerosis (FSGS).

Travere Therapeutics公司宣布，美国食品药品监督管理局（FDA）已接受其补充新药申请（sNDA），用于FILSPARI®（sparsetan）治疗局灶节段性肾小球硬化症（FSGS）。

FSGS is a rare kidney disorder that often leads to kidney failure. It affects both children and adults and is marked by protein leakage into the urine due to damage to the kidney's filtering units. This leakage can lead to further kidney damage. It is estimated that over 40,000 people in the United States are affected by FSGS, with similar numbers in Europe.

FSGS是一种罕见的肾脏疾病，常导致肾衰竭。它影响儿童和成人，其特征是由于肾脏过滤单位受损，蛋白质泄漏到尿液中。这种泄漏可能导致进一步的肾脏损伤。据估计，美国有超过40,000人受FSGS影响，欧洲也有类似数量的患者。

There are currently no approved pharmacological treatments specifically for FSGS..

目前尚无专门针对FSGS的获批药物治疗方法。

FILSPARI is an oral, non-immunosuppressive therapy that blocks both the endothelin A receptor (ETAR) and the angiotensin II subtype 1 receptor (AT1R), which are involved in podocyte injury – a key feature of FSGS. It is already approved to slow the progression of kidney damage in adults with IgA nephropathy..

FILSPARI 是一种口服、非免疫抑制疗法，可同时阻断内皮素 A 受体 (ETAR) 和血管紧张素 II 1 型受体 (AT1R)，这两种受体与足细胞损伤有关，而足细胞损伤是 FSGS 的关键特征。它已被批准用于减缓 IgA 肾病成人患者肾损伤的进展。

The application seeks traditional approval of the treatment and has been assigned a Prescription Drug User Fee Act (PDUFA) target action date of 13 January 2026. The FDA has also indicated plans to hold an advisory committee meeting to review the application.

该申请寻求对这种治疗方案的传统批准，并已设定《处方药使用者费用法案》（PDUFA）目标行动日期为2026年1月13日。FDA还表示计划召开一个咨询委员会会议来审查这项申请。

The sNDA submission is supported by data from the Phase 3 DUPLEX study and the Phase 2 DUET study. These are among the most comprehensive interventional studies carried out to date in FSGS, involving both adult and paediatric patients.

sNDA 提交得到了 3 期 DUPLEX 研究和 2 期 DUET 研究数据的支持。这些研究是迄今为止在 FSGS 领域进行的最全面的干预性研究之一，涵盖了成人和儿科患者。

Results from these studies demonstrated that FILSPARI led to rapid and sustained reductions in proteinuria compared to irbesartan, a commonly used treatment. The safety profile of FILSPARI was comparable to that of irbesartan, with no new safety concerns reported.

这些研究的结果表明，与常用于治疗的厄贝沙坦相比，FILSPARI 能够快速且持续地减少蛋白尿。FILSPARI 的安全性与厄贝沙坦相当，未报告新的安全隐患。

Patients completing the blinded phases of the DUPLEX and DUET studies were given the opportunity to join an open-label extension.

完成DUPLEX和DUET研究的双盲阶段的患者有机会加入开放标签扩展研究。

Travere’s progress with FILSPARI represents a potential new option for people living with FSGS, a condition for which there are currently no approved targeted therapies.

Travere公司在FILSPARI方面的进展代表了对于患有FSGS的人们的一个潜在新选择，目前该病症尚无获批的针对性疗法。

Source: travere.com

来源：travere.com