Abeona Therapeutics Inc. announced the FDA has approved Zevaskyn (pronounced as ‘ZEE-vah-skin’) (prademagene zamikeracel) gene-modified cellular sheets, also known as pz-cel, as the first and only autologous cell-based gene therapy for the treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB), a serious and debilitating genetic skin disease..

Abeona Therapeutics Inc. 宣布，FDA 已批准 Zevaskyn（发音为“ZEE-vah-skin”）(prademagene zamikeracel) 基因修饰细胞片，也称为 pz-cel，作为首个且唯一的自体细胞基因疗法，用于治疗成人和儿童隐性营养不良型大疱性表皮松解症 (RDEB) 患者的伤口，这是一种严重且致残的遗传性皮肤病。

There is no cure for RDEB and Zevaskyn is the only FDA-approved product to treat RDEB wounds with a single application.

RDEB目前尚无治愈方法，而Zevaskyn是唯一获得FDA批准的、可单次应用治疗RDEB伤口的产品。

“Today’s approval of Zevaskyn represents a pivotal moment in the treatment of RDEB, answering the call of people living with the clinical, economic, and human impact of this devastating disease,” said Vish Seshadri, Ph.D., M.B.A., Chief Executive Officer of Abeona. “We have heard from the RDEB community that there is a persistent unmet need to reliably address RDEB wounds, especially those that are chronic and prone to infection.

“今天对Zevaskyn的批准代表了RDEB治疗中的一个关键时刻，回应了那些承受着这种毁灭性疾病临床、经济和人性影响的患者的呼唤。”Abeona首席执行官Vish Seshadri博士、工商管理硕士说道，“我们从RDEB社区了解到，始终存在一个未满足的需求，即可靠地处理RDEB伤口，尤其是那些慢性且容易感染的伤口。”

Through a single surgical application, Zevaskyn  can now offer people with RDEB the opportunity for wound healing and pain reduction in even the most severe wounds, as evidenced by the results from our pivotal Phase 3 study.”.

通过单次手术应用，Zevaskyn现在可以为RDEB患者提供伤口愈合和疼痛减轻的机会，即使是最严重的伤口也不例外，我们关键的第三阶段研究结果已证明了这一点。"

First-of-its-kind gene therapy with robust body of clinical evidence The FDA approval of Zevaskyn is based on the pivotal Phase III VIITAL study (NCT04227106), a multi-center, randomized, intra-patient-controlled trial that met its two co-primary efficacy endpoints demonstrating statistically significant healing of 50 percent or more from baseline in large chronic RDEB wounds, and pain reduction from baseline as assessed by the Wong-Baker FACES scale, as evaluated at six months after treatment.

首个具有强大临床证据支持的基因疗法 Zevaskyn 的 FDA 批准基于关键的 III 期 VIITAL 研究（NCT04227106），这是一项多中心、随机、患者内对照试验，达到了两个共同主要疗效终点，证明在大型慢性 RDEB 伤口中，与基线相比有 50% 或以上的统计学显著愈合，并且根据 Wong-Baker FACES 量表评估，治疗后六个月时疼痛较基线显著减轻。

Across 43 large and chronic wounds treated with a single application of Zevaskyn, 81 percent of wounds showed 50 percent or more healing (P<0.0001) as evaluated at six months, compared to 16 percent in 43 matched control wounds treated with standard of care. The most common adverse events were observed in fewer than five percent of patients and included procedural pain and itch..

在使用Zevaskyn单次治疗的43个大型慢性伤口中，81%的伤口在六个月时显示出50%或更多的愈合（P<0.0001），而接受标准护理的43个匹配对照伤口中仅有16%。最常见的不良事件发生在不到5%的患者中，包括操作疼痛和瘙痒。

“Zevaskyn was well-tolerated and efficacious in clinical studies, providing clinically meaningful improvements in wound healing, pain reduction, and other associated symptoms in large chronic RDEB wounds after a single application,” said Jean Tang, M.D., Ph.D., professor of dermatology and lead principal investigator of the VIITAL study.

“Zevaskyn在临床研究中表现出良好的耐受性和有效性，单次应用后，在大型慢性RDEB伤口的愈合、疼痛减轻及其他相关症状方面均显示出具有临床意义的改善，”VIITAL研究的首席主要研究员、皮肤病学教授Jean Tang博士表示。

“In the completed Phase 1/IIa study of Zevaskn, we have observed wound healing and pain reduction that have lasted for years after a single application. Today we can celebrate the availability of an exciting new therapeutic option made possible by the incredible courage of patients and families who participated in these clinical studies.”.

“在Zevaskn的已完成的I/IIa期研究中，我们观察到单次应用后伤口愈合和疼痛减轻的效果持续了数年之久。今天，我们可以庆祝这一令人兴奋的新治疗选择得以实现，这要归功于参与这些临床研究的患者和家属们的非凡勇气。”

In the Phase 1/IIa study of Zevaskyn (NCT01263379), a single center, open label study in 38 chronic wounds across 7 patients showed that a single surgical application of Zevaskyn was associated with long-term improvement at treated sites over a median follow-up of 6.9 years; range 4 to 8 years.

在Zevaskyn（NCT01263379）的I/IIa期研究中，一项单中心、开放标签的研究针对7名患者的38处慢性伤口显示，单次手术应用Zevaskyn与治疗部位的长期改善相关，中位随访时间为6.9年；范围为4至8年。

Across both clinical studies, Zevaskyn was well-tolerated with no treatment-related serious adverse events observed to date.

在两项临床研究中，Zevaskyn 耐受性良好，迄今为止未观察到与治疗相关的严重不良事件。

Zevaskyn  is expected to be available beginning in the third quarter of 2025 through Zevaskyn  Qualified Treatment Centers (QTCs). The QTCs are well-recognized epidermolysis bullosa treatment centers with cell and gene therapy experience, situated across the U.S. to ensure patients nationwide have access to this important treatment..

预计Zevaskyn将于2025年第三季度开始通过Zevaskyn合格治疗中心（QTCs）提供。这些QTCs是广受认可的大疱性表皮松解症治疗中心，拥有细胞和基因治疗经验，分布在美国各地，以确保全国患者都能获得这一重要治疗。

Condition:

条件：

Epidermolysis Bullosa

表皮松解症

Type:

类型：

drug

药物