Travere Therapeutics has announced that the US Food and Drug Administration (FDA) has approved updated Risk Evaluation and Mitigation Strategy (REMS) labelling for FILSPARI® (sparsentan), the only dual endothelin angiotensin receptor antagonist indicated for the treatment of IgA nephropathy (IgAN).

Travere Therapeutics 宣布，美国食品药品监督管理局 (FDA) 已批准对 FILSPARI®（sparsentan）的更新风险评估与缓解策略 (REMS) 标签，这是唯一一种用于治疗 IgA 肾病 (IgAN) 的双重内皮素血管紧张素受体拮抗剂。

IgA nephropathy (IgAN), also known as Berger’s disease, is a rare progressive kidney disorder caused by the build-up of immunoglobulin A (IgA) in the kidneys. This disrupts normal kidney filtration and can lead to blood in the urine, proteinuria, swelling, high blood pressure, and progressive kidney damage.

IgA肾病（IgAN），也被称为Berger病，是一种罕见的进展性肾脏疾病，由免疫球蛋白A（IgA）在肾脏中积累引起。这会破坏正常的肾脏过滤功能，可能导致尿液中出现血液、蛋白尿、肿胀、高血压以及进行性肾脏损伤。

It is the most common form of primary glomerulonephritis globally and a leading cause of kidney failure, affecting up to 150,000 people in the United States and significant numbers in Europe and Japan..

它是全球最常见的原发性肾小球肾炎形式，也是导致肾衰竭的主要原因，影响美国多达15万人，同时在欧洲和日本也有大量患者。

Focal segmental glomerulosclerosis (FSGS) is a rare condition that causes scarring in the kidneys and affects both adults and children. It is estimated to impact more than 40,000 people in the United States, with similar prevalence in Europe. The condition is marked by proteinuria, which damages kidney function, and can lead to swelling, low blood albumin, abnormal lipid levels, high blood pressure, and eventually kidney failure.

局灶性节段性肾小球硬化（FSGS）是一种罕见的疾病，会导致肾脏瘢痕形成，影响成人和儿童。据估计，美国有超过40,000人受此疾病影响，欧洲的患病率与此类似。该病的特征是蛋白尿，会损害肾功能，并可能导致水肿、低血白蛋白、血脂异常、高血压，最终引发肾衰竭。

Currently, there are no FDA-approved pharmacological treatments for FSGS, and sparsentan is not yet approved for this indication..

目前，尚无FDA批准的FSGS药物治疗方法，且sparsentan尚未获批用于此适应症。

The revised guidance reduces the frequency of liver function monitoring from monthly to once every three months, starting from the initiation of treatment. In addition, the requirement for embryo-foetal toxicity monitoring has been removed. These changes are based on safety data from post-marketing surveillance and findings from the Phase 3 PROTECT trial in IgAN, the Phase 3 DUPLEX trial, and the Phase 2 DUET study in focal segmental glomerulosclerosis (FSGS)..

修订后的指南将治疗开始后的肝功能监测频率从每月一次减少到每三个月一次。此外，胚胎-胎儿毒性监测的要求已被取消。这些更改基于上市后监测的安全数据，以及IgAN的3期PROTECT试验、3期DUPLEX试验和局灶节段性肾小球硬化症（FSGS）的2期DUET研究的结果。

The FDA decision to remove the embryo-foetal toxicity monitoring requirement followed an assessment of pregnancy data gathered over the past two decades from the use of endothelin receptor antagonist medicines.

FDA决定取消胚胎-胎儿毒性监测要求，这是基于过去二十年中内皮素受体拮抗剂药物使用所收集的妊娠数据的评估结果。

A supplemental New Drug Application (sNDA) for FILSPARI in FSGS is under review, with a Prescription Drug User Fee Act (PDUFA) action date set for 13 January 2026. If approved, FILSPARI would become the first and only therapy available for this condition.

FILSPARI用于治疗FSGS的补充新药申请（sNDA）正在审查中，处方药使用者费用法案（PDUFA）的决定日期定为2026年1月13日。如果获得批准，FILSPARI将成为针对该疾病的首个也是唯一的疗法。

Source: travere.com

来源：travere.com