New Data Strengthens Long-Term Safety Profile of DUVYZAT for Duchenne muscular dystrophy

New Data Strengthens Long-Term Safety Profile of DUVYZAT for Duchenne muscular dystrophy

By, Admin 10 March 2026

New research presented at a major medical conference provides deeper insight into the long-term safety and clinical impact of the Duchenne muscular dystrophy therapy DUVYZAT (givinostat).

ITF Therapeutics LLC, the U.S. rare-disease affiliate of Italfarmaco S.p.A., presented 10 scientific abstracts at the Muscular Dystrophy Association Clinical and Scientific Conference 2026, held March 8–11, 2026.

The findings include long-term safety observations and new analyses related to muscle preservation and mobility outcomes in patients with Duchenne muscular dystrophy (DMD).

Long-Term Safety Data from Ongoing Study

One of the key presentations focused on interim safety results from an open-label extension study involving patients who previously participated in givinostat clinical trials. The extension follows participants from the Phase 3 EPIDYS trial.

Key highlights include:

  • Long-term safety monitoring of givinostat treatment
  • Data collected through December 31, 2023
  • Participants who completed or were screened for earlier givinostat studies

Researchers continue to track treatment outcomes as patients remain on therapy.

Evidence of Slower Muscle Degeneration

Another analysis examined how givinostat affects muscle composition and function in DMD. Researchers evaluated:

  • Contractile cross-sectional area (cCSA) — a measure of functional muscle tissue
  • Fat fraction — a marker of muscle degeneration

Using magnetic resonance imaging and spectroscopy, investigators observed that givinostat:

  • Reduced the decline in contractile muscle area
  • Lowered fat infiltration in muscle tissue

These measures are important indicators of disease progression in DMD.

Potential Delay in Loss of Walking Ability

A separate analysis explored the impact of long-term givinostat therapy on loss of ambulation, a key milestone in DMD progression.

The study evaluated:

  • Patients aged 6 years and older
  • Givinostat used alongside standard corticosteroid therapy
  • Additional follow-up data extending two years beyond earlier analyses

Results suggest treatment may delay the age at which patients permanently lose the ability to walk independently.

New Insights into Disease Biology

Researchers also presented laboratory data showing how givinostat may influence the underlying biology of DMD. The study analyzed how the drug affects fibrosis pathways triggered by TGF-β signaling in muscle-supporting cells.

Using genomic tools such as RNA-seq and ATAC-seq, scientists found that givinostat can:

  • Reprogram fibrotic gene networks
  • Reduce pathological stromal cell activity
  • Potentially restore healthier muscle-repair conditions

Experts Emphasize Importance of Long-Term Data

Scott Baver, Vice President and Head of Global Medical Affairs for Rare Diseases at Italfarmaco, said the findings help expand understanding of the therapy.

“By further evaluating safety, efficacy outcomes, and dosing considerations, we aim to support informed treatment decisions for people living with DMD.”

Clinical experts also highlighted the value of extended follow-up data. Aravindhan Veerapandiyan of the University of Arkansas for Medical Sciences and Arkansas Children’s Hospital noted that continued analysis is important as clinicians personalize treatment strategies for patients with DMD.

About DUVYZAT (Givinostat)

DUVYZAT is an FDA-approved histone deacetylase (HDAC) inhibitor for patients aged six years and older with Duchenne muscular dystrophy.

HDAC enzymes regulate muscle repair and inflammation. In DMD, these enzymes become overactive, contributing to:

  • Chronic muscle inflammation
  • Reduced muscle regeneration
  • Replacement of muscle tissue with fat and scar tissue

Givinostat works by inhibiting HDAC activity, which may help slow muscle degeneration and improve the body’s ability to repair muscle tissue.

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