Sangamo Therapeutics, Inc. announced the outcome of a recent successful interaction with the  FDA, providing a clear regulatory pathway to Accelerated Approval for isaralgagene civaparvovec, or ST 920, its wholly owned gene therapy product candidate for the treatment of Fabry disease

Sangamo Therapeutics，Inc.宣布了最近与FDA成功互动的结果，为加速批准isaralgagene civaparvovec（或ST 920）提供了明确的监管途径，ST 920是其全资拥有的用于治疗法布里病的候选基因治疗产品

The FDA has agreed in a Type B interaction that data from the ongoing Phase 1/II STAAR study can serve as the primary basis for approval under the Accelerated Approval Program, using eGFR slope at 52 weeks across all patients as an intermediate clinical endpoint. The complete dataset to support an Accelerated Approval pathway will be available in the first half of 2025.

FDA在B型相互作用中同意，正在进行的1/II期STAAR研究的数据可以作为加速批准计划下批准的主要基础，使用所有患者52周的eGFR斜率作为中间临床终点。支持加速审批途径的完整数据集将于2025年上半年提供。

This approach unlocks a potential BLA submission in the second half of 2025, three years ahead of previous estimates, and avoids the requirement for an additional registrational study to establish clinical efficacy..

这种方法在2025年下半年解锁了潜在的BLA提交，比以前的估计提前了三年，并且避免了需要额外的注册研究来确定临床疗效。。

Sangamo engaged with the FDA on alternative pathways to potential approval following analysis of clinical data from the Phase 1/II STAAR study showing encouraging safety and efficacy data, including promising preliminary evidence of improved kidney function. Renal manifestations, such as proteinuria or a decreased glomerular filtration rate, occur early in life in almost all male, and in many female, patients with Fabry disease, and can lead to end-stage renal disease and early death.

Sangamo在对1/II期STAAR研究的临床数据进行分析后，与FDA就潜在批准的替代途径进行了接触，这些数据显示了令人鼓舞的安全性和有效性数据，包括有希望的肾功能改善的初步证据。几乎所有男性和许多女性法布里病患者在生命早期都会出现肾脏表现，例如蛋白尿或肾小球滤过率降低，并可能导致终末期肾脏疾病和早期死亡。

In the 18 male and female patients treated with isaralgagene civaparvovec with more than one year of follow-up data, statistically significant improvements were observed in both mean and median eGFR levels, resulting in a positive annualized eGFR slope. Based on these latest data, the FDA agreed that eGFR slope at 52 weeks can serve as an intermediate clinical endpoint to support a potential Accelerated Approval.

在接受isaralgagene civaparvovec治疗的18名男性和女性患者中，随访数据超过一年，平均和中位eGFR水平均观察到统计学上显着的改善，导致eGFR年化斜率为正。根据这些最新数据，FDA同意52周时的eGFR斜率可以作为中间临床终点，以支持潜在的加速批准。

The FDA also advised that eGFR slope at 104 weeks may be assessed to verify clinical benefit..

FDA还建议可以评估104周时的eGFR斜率，以验证临床益处。。

“Fabry is a debilitating disease, for which there is a serious unmet medical need,” said Sandy Macrae, Chief Executive Officer of Sangamo. “I strongly believe in the potential for ST 920 to alleviate many manifestations of Fabry disease and am delighted to have a clear regulatory pathway that could bring this treatment to patients significantly sooner than originally anticipated”..

Sangamo首席执行官桑迪·麦克雷（SandyMacrae）表示：“法布里（Fabry）是一种使人衰弱的疾病，严重的医疗需求尚未得到满足。”。“我坚信ST 920有可能缓解法布里病的许多表现，并且很高兴有一个明确的监管途径，可以比最初预期的更快地为患者带来这种治疗”。。

Dosing was completed in the Phase 1/II STAAR study in April 2024, with a total of 33 patients dosed. The longest treated patient recently achieved four years of follow-up. The 18th and final patient who started the study on Enzyme Replacement Therapy (ERT) was successfully withdrawn from ERT in September 2024, and all 18 patients remain off ERT as of today.

2024年4月，在1/II期STAAR研究中完成了给药，共有33名患者给药。最近接受治疗时间最长的患者获得了四年的随访。2024年9月，开始进行酶替代疗法（ERT）研究的第18位也是最后一位患者成功退出了ERT，截至今日，所有18位患者均未接受ERT治疗。

The 52-week eGFR slope data from all enrolled patients in the Phase 1/II STAAR study will be available in the first half of 2025. A potential BLA submission is anticipated in the second half of 2025..

2025年上半年将提供1/II期STAAR研究中所有入选患者的52周eGFR斜率数据。。。

Condition: Fabry Disease

病情：法布里病

Type: drug

类型：药物