Apellis Pharmaceuticals has received approval from the US Food and Drug Administration (FDA) for EMPAVELI® (pegcetacoplan) as the first treatment for C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in patients aged 12 years and older.

Apellis制药公司已获得美国食品和药物管理局（FDA）批准，将EMPAVELI®（pegcetacoplan）作为首个用于治疗12岁及以上患者的C3肾小球病（C3G）和原发性免疫复合物膜增生性肾小球肾炎（IC-MPGN）的药物。

C3G and primary IC-MPGN are progressive and often result in kidney failure, typically within five to ten years of diagnosis. Many patients eventually require lifelong dialysis or kidney transplantation. Disease recurrence is also common following transplantation.

C3G和原发性IC-MPGN病情会逐步进展，通常在诊断后五年到十年内发展为肾衰竭。许多患者最终需要终身透析或肾移植。移植后疾病复发也很常见。

EMPAVELI is the first therapy approved to target excessive C3 activation, a key contributor to the inflammation and damage associated with these diseases. It is administered twice weekly and is designed to regulate the complement cascade, part of the body’s immune system.

EMPAVELI 是首个获准用于靶向过度 C3 激活的疗法，C3 过度激活是这些疾病相关炎症和损伤的主要因素。该药物每周两次给药，旨在调节补体级联反应，这是人体免疫系统的一部分。

The safety profile of the treatment is supported by over 2,200 patient-years of data. The most common side effects reported in the VALIANT study were infusion site reactions, fever, nasopharyngitis, flu, cough, and nausea.

该治疗的安全性特征得到了超过2200患者年的数据支持。VALIANT研究中报告的最常见的副作用是输注部位反应、发热、鼻咽炎、流感、咳嗽和恶心。

EMPAVELI is already approved in several markets for the treatment of paroxysmal nocturnal haemoglobinuria (PNH) and is being further explored for other rare conditions.

EMPAVELI已在多个市场获批用于治疗阵发性睡眠性血红蛋白尿症（PNH），并正在进一步探索用于其他罕见病。

These rare kidney diseases are estimated to affect around 5,000 people in the United States.

这些罕见的肾脏疾病据估计会影响美国大约5000人。

The approval is based on positive results from the Phase 3 VALIANT study, which demonstrated significant improvements across key disease markers. EMPAVELI treatment led to a 68% reduction in proteinuria compared with placebo.

该批准基于三期VALIANT研究的积极结果，该研究表明在关键疾病标志物方面取得了显著改善。与安慰剂相比，EMPAVELI治疗使蛋白尿减少了68%。

In addition, patients showed stabilised kidney function and a marked reduction in C3 deposits. These outcomes were observed across both adolescent and adult patients, including those with post-transplant recurrence.

此外，患者的肾功能稳定，C3沉积显著减少。这些结果在青少年和成人患者中均有观察到，包括移植后复发的患者。

The VALIANT study involved 124 patients aged 12 years and older and is the largest clinical trial to date in these populations. Participants were randomised to receive either EMPAVELI or a placebo over 26 weeks.

VALIANT 研究涉及 124 名 12 岁及以上的患者，是迄今为止在这些人群中规模最大的临床试验。参与者被随机分配在 26 周内接受 EMPAVELI 或安慰剂。

A further open-label phase followed, allowing all patients access to EMPAVELI.

随后是一个进一步的开放标签阶段，允许所有患者使用EMPAVELI。