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<p>Givinostat (Duvyzat<sup>™</sup>) is an efficacious and generally well-tolerated histone deacetylase (HDAC) inhibitor approved in the EU and the USA for the treatment of Duchenne muscular dystrophy (DMD) in patients aged ≥ 6&#xa0;years. It is the first non-steroidal therapy available for the treatment of DMD arising from all genetic causes. By inhibiting HDACs, some of which are constitutively activated in DMD, givinostat initiates a cascade of events leading to improved muscle regeneration and reduced inflammation in dystrophin-deficient muscles. In the randomised, multinational phase&#xa0;3 EPIDYS trial in ambulant boys with DMD receiving concomitant corticosteroid treatment, twice-daily oral givinostat significantly reduced four-stair climb worsening compared with placebo over 72&#xa0;weeks. Additional functional outcomes favoured givinostat over placebo but did not reach statistical significance. The most common adverse events were thrombocytopenia, hypertriglyceridaemia and gastrointestinal disturbances; these were typically mild or moderate in severity and were generally manageable with dose reduction or interruption. The ongoing long-term safety extension has not identified any new safety signals.</p>

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Givinostat in Duchenne muscular dystrophy: a profile of its use

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2026年4月21日

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  • Aisling McGuigan,
  • Matt Shirley

摘要

Givinostat (Duvyzat™) is an efficacious and generally well-tolerated histone deacetylase (HDAC) inhibitor approved in the EU and the USA for the treatment of Duchenne muscular dystrophy (DMD) in patients aged ≥ 6 years. It is the first non-steroidal therapy available for the treatment of DMD arising from all genetic causes. By inhibiting HDACs, some of which are constitutively activated in DMD, givinostat initiates a cascade of events leading to improved muscle regeneration and reduced inflammation in dystrophin-deficient muscles. In the randomised, multinational phase 3 EPIDYS trial in ambulant boys with DMD receiving concomitant corticosteroid treatment, twice-daily oral givinostat significantly reduced four-stair climb worsening compared with placebo over 72 weeks. Additional functional outcomes favoured givinostat over placebo but did not reach statistical significance. The most common adverse events were thrombocytopenia, hypertriglyceridaemia and gastrointestinal disturbances; these were typically mild or moderate in severity and were generally manageable with dose reduction or interruption. The ongoing long-term safety extension has not identified any new safety signals.