TUESDAY, April 29, 2025 -- The U.S. Food and Drug Administration has approved Zevaskyn (prademagene zamikeracel) for patients with the rare, genetic skin disorder recessive dystrophic epidermolysis bullosa.

2025年4月29日，星期二 —— 美国食品药品监督管理局已批准Zevaskyn（prademagene zamikeracel）用于治疗罕见的遗传性皮肤病——隐性营养不良型大疱性表皮松解症患者。

Zevaskyn is the first and only autologous cell-based gene therapy for the treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa, a skin condition characterized by extremely fragile skin that blisters and tears easily.

Zevaskyn 是首个也是唯一一个用于治疗患有隐性营养不良性大疱性表皮松解症的成人和儿童患者伤口的自体细胞基因疗法，这种皮肤病的特征是皮肤极其脆弱，容易起水泡和撕裂。

FDA approval of Zevaskyn was based on findings from two clinical studies. In a single-center, open-label, phase 1/2a study involving 38 chronic wounds in seven patients, a single surgical application of Zevaskyn demonstrated long-term improvement at the treated sites during a median seven-year follow-up period..

FDA 批准 Zevaskyn 是基于两项临床研究的结果。在一项单中心、开放标签、1/2a 期研究中，涉及 7 名患者的 38 处慢性伤口，单次手术应用 Zevaskyn 在中位七年随访期间显示出治疗部位的长期改善。

In the multicenter, randomized, intrapatient-controlled, phase 3 VIITAL study, evaluating the effectiveness of a single application of Zevaskyn on 43 large, chronic wounds, 81 percent of wounds demonstrated 50 percent or greater healing after six months. This result was statistically significant when compared with a matched control group of 43 wounds treated with the standard of care, where only 16 percent achieved the same level of healing..

在多中心、随机、患者内对照的第三阶段VIITAL研究中，评估了Zevaskyn单次应用于43个大型慢性伤口的效果，81%的伤口在六个月后显示出50%或更高的愈合率。这一结果与接受标准治疗的43个伤口的匹配对照组相比具有统计学意义，对照组中仅有16%达到了相同的愈合水平。

Across both studies, Zevaskyn was well tolerated, and no treatment-related serious adverse events were reported. The most common nonserious adverse events reported were procedural pain and itch, which only appeared to affect about 5 percent of patients.

在两项研究中，Zevaskyn 耐受性良好，未报告与治疗相关的严重不良事件。最常见的非严重不良事件是手术疼痛和瘙痒，似乎仅影响约 5% 的患者。

'Based on the strength of our data across clinical trials, we are confident in Zevaskyn's ability to deliver long-term results after a single treatment application,' Madhav Vasanthavada, Ph.D., chief commercial officer at Abeona Therapeutics, said in a statement. 'We are committed to working closely with both commercial and government payers on outcome-based agreements that stand behind the promise of Zevaskyn for patients, and expedite access.'.

“基于我们在临床试验中的数据优势，我们对Zevaskyn在单次治疗后提供长期效果的能力充满信心，”Abeona Therapeutics首席商务官Madhav Vasanthavada博士在一份声明中表示。“我们致力于与商业和政府支付方密切合作，制定基于结果的协议，兑现Zevaskyn对患者的承诺，并加速获取途径。”

Approval of Zevaskyn was granted to Abeona Therapeutics.

Zevaskyn的批准已授予Abeona Therapeutics。

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