Efficacy of Givinostat by Age (6–7 and >7 years): A Post Hoc Analysis of EPIDYS

Thursday, October 23, 2025

3:45 PM - 5:00 PM MT

Location: Session Room - 250 DEF

Primary Author(s)

Chamindra Laverty, MD (she/her/hers)

Clinical Professor of Neurosciences
University of California, San Diego
San Diego, California

Co-Author(s)

FA

Federica Alessi, Other (she/her/hers)

Biometrics and Data Sciences Director
Italfarmaco SPA
Milan, Lombardia, Italy
NC

Nicoletta Coceani, Other

Clinical scientist
Italfarmaco SpA
Milan, Lombardia, Italy
PB

Paolo Bettica, MD, PhD

Chief Medical Officer
Italfarmaco SpA.
Milan, Italy
DV

Dragana Vucinic, MD

Child neurologist
Clinic of Neurology and Psychiatry for Children and Youth, Belgrade, Serbija
Beograd, Serbia

Disclosure(s):

Chamindra Laverty, MD: No relevant disclosure to display

Dragana Vucinic, MD: No financial relationships to disclose

Background and/or Objectives: The effect of givinostat was investigated in a double-blind, randomized, phase 3 EPIDYS study (NCT02851797) in patients 6 years of age or older. Natural history data suggest that functional decline in DMD becomes more pronounced around 7 years of age.

Design: These post hoc analyses of EPIDYS evaluated the efficacy of givinostat in patients aged 6–7 and >7 years at baseline.

Setting : 41 tertiary care sites in 11 countries.

Participants : Patients from EPIDYS.

Interventions: 81 patients received givinostat (20 patients aged 6–7 years, 61 patients aged >7 years), and 39 patients received placebo (7 patients aged 6–7 years, 32 patients aged >7 years).

Main Outcome Measures: Mean change from baseline to 72 weeks in 4-stair climb (4SC) time (seconds) and total North Star Ambulatory Assessment (NSAA) score. Data were analyzed using analysis of covariance.

Results: At 72 weeks, the geometric least squares (LS) mean ratio (givinostat/placebo) for log-transformed 4SC versus baseline was 0.69 (95% CI, 0.49-0.99) for patients aged 6–7 years and 0.88 (0.76-1.03) for patients aged >7 years. Using log-transformed data, the geometric LS mean change from baseline in 4SC time was 1.17 (1.00-1.38) for givinostat vs 1.70 (1.26-2.27) for placebo in patients aged 6–7 years and 1.29 (1.18-1.41) for givinostat vs 1.46 (1.29-1.65) for placebo in patients aged >7 years. The LS mean difference between groups in NSAA total score was 3.77 (−0.76 to 8.29) in patients aged 6–7 years and 1.32 (−0.50 to 3.13) in patients aged >7 years.

Conclusions: Givinostat showed delayed disease progression versus placebo in patients aged 6–7 years and >7 years, further supporting its consistent benefit in patients with DMD.