Duvyzat® efficacy1
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EPIDYS study: Primary endpoint
Patients on Duvyzat® experienced lower motor function decline measured by 4SC than control group
- Significant difference between Duvyzat® and control from Week 48 through to Week 72
EPIDYS: Observed mean change to complete 4SC test
At the end of the 18-month study period, the log-transformed 4SC GLS mean ratio vs baseline was 1.27 (95% CI: 1.17, 1.37) for Duvyzat® and 1.48 (95% CI: 1.32, 1.66) for the control group (ratio 0.86 [95% CI: 0.745, 0.989], p = 0.035).
Data are mean (95% CI). The confidence intervals have not been adjusted for multiplicity and should not be used for hypothesis testing systemic corticosteroids, in a dose and regimen that was to remain unchanged over the follow-up period. Baseline mean values were 3.39 and 3.48 s for the Duvyzat® and control groups, respectively.
All patients were also receiving systemic corticosteroids, in a dose and regimen that was to remain unchanged over the follow-up period.
EPIDYS study: Secondary endpoints
- All key secondary endpoints favoured Duvyzat® vs control, although no statistical significance was reached
- Over time, Duvyzat® consistently reduced decline in muscle function and strength as well as fat infiltration
EPIDYS: Observed mean change in NSAA total score from baseline (key secondary endpoint)
- The difference from baseline to Week 72 in NSAA total score between the two groups was -1.91 (p = 0.020)
Patients on Duvyzat® experience a lower functional loss over time than control group2
NSAA cumulative loss of function over 72 weeks (key secondary endpoint)
- Patients treated with Duvyzat® failed less NSAA items than patients in control group at any time point
- The difference from baseline to Week 72 in NSAA cumulative loss of function between the two groups was 2.14
Mean change from baseline over 72 weeks in muscle groups fat fraction
- By reducing fat infiltration, Duvyzat® preserves muscle function
- VL was the key secondary endpoint1. BFLH, ST, quadriceps, and hamstrings were exploratory endpoints3
Adverse events should be reported. Reporting forms and information can be found at https://yellowcard.mhra.gov.uk/ or search for MHRA Yellow Card in the Google Play or Apple App Store.
Adverse events should also be reported to ITF Pharma Ltd Medical Information on: 0800 098 4040 or UK.Medical.Information@italfarmacogroup.com
Product Quality Complaints should also be reported to ITF Pharma Ltd Medical Information on: 0800 098 4040 or UK.Medical.Information@italfarmacogroup.com
References
- Mercuri E, Vilchez JJ, Boespflug-Tanguy O, et al. Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Neurol. 2024; 23: 393-403.
- Mercuri E, Vilchez JJ, Boespflug-Tanguy O, et al. Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Neurol. 2024; 23: 393-403. Supplementary Appendix.
- Vandenborne K, et al. Oral presentation at Muscular Dystrophy Association Clinical & Scientific Conference; 19–22 March 2023; Dallas, TX, USA.
