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Background <p>Patients with dystrophic epidermolysis bullosa have pathogenic variants in <i>COL7A1</i>, leading to skin fragility. Beremagene geperpavec-svdt (B-VEC) is a modified, herpes simplex virus&#xa0;type&#xa0;1-based gene therapy vector that topically delivers <i>COL7A1</i> to dystrophic epidermolysis bullosa wounds. In a phase III study, B-VEC significantly improved wound healing at 3 and 6 months compared with placebo.</p> Objective <p>We aimed to evaluate the safety and tolerability of B-VEC beyond 6 months in patients with dystrophic epidermolysis bullosa.</p> Methods <p>An open-label extension study was conducted with 47 subjects (24 rollover from phase III; 23 treatment naïve) receiving B-VEC weekly to target wound areas for up to 112 weeks (median 81 weeks). Safety was assessed by adverse events. Treatment satisfaction and quality of life were assessed with patient-reported outcomes as exploratory measures of efficacy. Selected wounds from phase III rollover subjects were assessed for closure.</p> Results <p>Thirty-five subjects (74.5%) reported one or more adverse events; most were mild or moderate in severity. Fourteen subjects experienced 17 serious adverse events and ten experienced 14 severe adverse events; none was considered treatment related. No adverse events led to treatment or study discontinuation. Patient-reported outcomes indicated high levels of treatment satisfaction, but were inconclusive with regard to quality of life. Among rollover subjects, wounds that received B-VEC during phase III maintained high closure rates during the open-label extension (range 61.1–89.5%, assessed baseline to month 12).</p> Limitations <p>This was an open-label design, with a variable follow-up.</p> Conclusions <p>Patients undergoing extended B-VEC treatment maintained high satisfaction and continued to respond to treatment with no new safety signals detected in the open-label extension study, supporting the continuous use of B-VEC.</p> Clinical Trial Registration <p>NCT04917874 (date of trial registration: 8 June, 2021).</p>

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Long-Term Safety and Tolerability of Beremagene Geperpavec-svdt (B-VEC) in an Open-Label Extension Study of Patients with Dystrophic Epidermolysis Bullosa

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2025年4月12日

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  • M. Peter Marinkovich,
  • Amy S. Paller,
  • Shireen V. Guide,
  • Mercedes E. Gonzalez,
  • Anne W. Lucky,
  • Işın Sinem Bağcı,
  • Brittani Agostini,
  • Kolleen Fitzgerald,
  • Shijie Chen,
  • Hubert Chen,
  • Meghan M. Conner,
  • Suma M. Krishnan

摘要

Background

Patients with dystrophic epidermolysis bullosa have pathogenic variants in COL7A1, leading to skin fragility. Beremagene geperpavec-svdt (B-VEC) is a modified, herpes simplex virus type 1-based gene therapy vector that topically delivers COL7A1 to dystrophic epidermolysis bullosa wounds. In a phase III study, B-VEC significantly improved wound healing at 3 and 6 months compared with placebo.

Objective

We aimed to evaluate the safety and tolerability of B-VEC beyond 6 months in patients with dystrophic epidermolysis bullosa.

Methods

An open-label extension study was conducted with 47 subjects (24 rollover from phase III; 23 treatment naïve) receiving B-VEC weekly to target wound areas for up to 112 weeks (median 81 weeks). Safety was assessed by adverse events. Treatment satisfaction and quality of life were assessed with patient-reported outcomes as exploratory measures of efficacy. Selected wounds from phase III rollover subjects were assessed for closure.

Results

Thirty-five subjects (74.5%) reported one or more adverse events; most were mild or moderate in severity. Fourteen subjects experienced 17 serious adverse events and ten experienced 14 severe adverse events; none was considered treatment related. No adverse events led to treatment or study discontinuation. Patient-reported outcomes indicated high levels of treatment satisfaction, but were inconclusive with regard to quality of life. Among rollover subjects, wounds that received B-VEC during phase III maintained high closure rates during the open-label extension (range 61.1–89.5%, assessed baseline to month 12).

Limitations

This was an open-label design, with a variable follow-up.

Conclusions

Patients undergoing extended B-VEC treatment maintained high satisfaction and continued to respond to treatment with no new safety signals detected in the open-label extension study, supporting the continuous use of B-VEC.

Clinical Trial Registration

NCT04917874 (date of trial registration: 8 June, 2021).