Givinostat Shows Long-Term Benefits for Duchenne Muscular Dystrophy

Givinostat Shows Long-Term Benefits for Duchenne Muscular Dystrophy

By, Admin 26 August 2025

For families facing Duchenne muscular dystrophy (DMD), time is everything. A new study brings hope—showing that givinostat can slow disease progression over years.

The Study at a Glance

ITF Therapeutics, the U.S. arm of Italfarmaco, released long-term safety and efficacy data on givinostat in Annals of Clinical and Translational Neurology.

The findings come from open-label extensions of Phase 2 and Phase 3 EPIDYS trials. Some patients have now been on givinostat for more than eight years.

Key highlights:

  • Patients treated with givinostat delayed loss of mobility functions by years compared to untreated peers.
  • Benefits included rising from the floor, climbing stairs, and walking.
  • The drug was well-tolerated, with no new safety concerns.

Numbers That Matter

When matched against a natural history cohort, givinostat patients showed:

  • 2 years longer before losing the ability to rise from the floor.
  • 3.3 years longer before losing the ability to climb stairs.
  • 2.9 years longer before losing the ability to walk.

For DMD patients, these gains mean more independence, more daily participation, and more time with loved ones.

Expert Voices

Dr. Paolo Bettica, Chief Medical Officer at Italfarmaco, emphasized the sustained benefits across mobility outcomes. He called givinostat a therapy capable of meaningfully altering the course of DMD.

Scott Baver, VP of Medical Affairs at ITF Therapeutics, noted the impact on family life: more time for patients to engage in the activities that matter most.

Approved and Accessible

Givinostat, marketed as Duvyzat, has received approvals in the U.S., EU, and U.K. for ambulant patients aged six and older.

  • FDA approval: March 2024.
  • U.K. MHRA approval: December 2024.
  • EU conditional approval: June 2025.

The drug is taken orally, in combination with corticosteroids. Its mechanism targets excessive histone deacetylase (HDAC) activity in muscle cells—restoring gene expression critical for muscle repair.

Why This Is Big

DMD is one of the most severe childhood neuromuscular disorders. Symptoms appear between ages two and five. Most boys lose the ability to walk in early adolescence. Eventually, the disease affects the heart and lungs.

By slowing progression, givinostat could shift the trajectory of this condition, giving patients and families something they have rarely had—extra years of mobility and independence.

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