Givinostat Efficacy & Safety: Long-Term Data in DMD
Givinostat Delays DMD Progression: At-a-Glance
What: A study published in annals of Clinical and Translational Neurology demonstrates that givinostat (Duvyzat; ITF Therapeutics), combined with corticosteroids, delays disease progression in Duchenne Muscular Dystrophy (DMD).
Were: The study analyzed data from open-label extensions of Phase 2 and Phase 3 (EPIDYS; NCT03373968) clinical trials. Data was compared to a natural history cohort.
When: Findings were published in 2024, based on data with maximum exposure to givinostat exceeding 8 years (including prior studies).
Why it Matters: givinostat offers a potential new treatment option for DMD, a devastating genetic disorder. The study shows sustained benefits in key mobility functions (rising from the floor, climbing stairs, ambulation) and a well-established safety profile. It suggests the drug can meaningfully alter the disease course.
What’s Next: Continued monitoring of long-term outcomes and further research to optimize treatment strategies with givinostat. Potential for broader adoption as a valuable treatment option for DMD patients.
Key Data:
| Functional Outcome | Median Delay with Givinostat (vs. Natural History) | HR (95% CI) | Nominal p-value |
|---|---|---|---|
| Loss of Ability to Rise from Floor | 2 years | 0.66 (0.45-0.96) | 0.028 |
| Loss of 4-Stair Climb Ability | 3.3 years | 0.39 (0.24-0.65) | < 0.001 |
| Loss of Ambulation | 2.9 years | 0.42 (0.2-0.76) | 0.004 |
Study Details:
Study Design: Open-label extension of Phase 2/3 trials, propensity-matched to a natural history cohort.
Participants: 142 patients receiving givinostat, 142 in the natural history cohort (after matching).
Mean Givinostat Exposure: 559.6 days. Drug: Givinostat (a class I and II histone deacetylase inhibitor).
* Primary Outcome: Delay in loss of key motor functions.