APPLY-PNH extension data show that continuous Fabhalta® (iptacopan) treatment in adults with paroxysmal nocturnal hemoglobinuria (PNH) enabled sustained hemoglobin-level increases to near-normal (≥12 g/dL), blood transfusion avoidance, and improved patient-reported fatigue in the majority of patients, with a safety profile consistent with previously reported data1-5Patients switching from anti-C5s to Fabhalta in the extension period achieved outcomes comparable to the Fabhalta arm in the 24-week randomized controlled period, including transfusion avoidance and near-normal hemoglobin-levels (≥12 g/dL) in the majority of patients1 Fabhalta was recently approved by the FDA for adults with PNH, including for both previously treated and treatment-naive patients6 Basel, December 11, 2023 – Novartis today announced results from the extension period of the pivotal Phase III APPLY-PNH trial of oral monotherapy Fabhalta® (iptacopan) in adults with paroxysmal nocturnal hemoglobinuria (PNH) who had residual anemia (hemoglobin <10 g/dL) despite previous anti-C5 therapy1,2.

APPLY-PNH扩展数据显示，对阵发性夜间血红蛋白尿（PNH）的成年人进行持续Fabhalta®（iptacopan）治疗可使持续的血红蛋白水平升高至接近正常（≥12 g/dL），避免输血，并改善大多数患者报告的疲劳，安全性与先前报道的数据一致1-5在延长期内从抗C5s转为Fabhalta的患者在24周的随机对照期内取得了与Fabhalta组相当的结果，包括大多数患者避免输血和接近正常的血红蛋白水平（≥12 g/dL）1 Fabhalta最近被FDA批准用于PNH成人，包括之前接受过治疗和未接受过治疗的患者6巴塞尔，2023年12月11日-诺华今天宣布了口服单一疗法Fabhalta®（iptacopan）的关键III期APPLY-PNH试验延长期的结果，该试验适用于阵发性夜间血红蛋白尿（PNH）患者，尽管之前接受过抗C5治疗，但仍有残余贫血（血红蛋白<10 g/dL）1,2。

Continuous Fabhalta treatment (200 mg twice daily) for 48 weeks enabled sustained hemoglobin-level increases to near-normal (12 g/dL or more), blood transfusion avoidance, and reduced patient-reported fatigue in the majority of patients; comparable benefits emerged in those patients switching from anti-C5 therapy to Fabhalta in the extension1.

持续Fabhalta治疗（200 mg，每日两次），持续48周，使血红蛋白水平持续升高至接近正常（12 g/dL或更高），避免输血，并减少大多数患者报告的疲劳；在扩展过程中，从抗C5治疗转向Fabhalta的患者出现了类似的益处1。

Data will be presented at the 65th American Society of Hematology Annual Meeting & Exposition (ASH).  “The new APPLY-PNH data are an expansion of the robust outcomes we saw in the randomized phase and demonstrate that patients with PNH who took Fabhalta experienced meaningful hemoglobin improvement over the longer term – nearly a year,” said principal co-investigator Anton.

数据将在第65届美国血液学会年会和博览会（ASH）上公布。首席联合研究员安东（Anton）说：“新的APPLY-PNH数据扩大了我们在随机阶段看到的稳健结果，并证明服用法巴他（Fabhalta）的PNH患者在较长期（近一年）内经历了有意义的血红蛋白改善。”。