Duvyzat® efficacy & safety
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PRESERVES
Muscle Function*1-4
DELAYS
Disease Progression*1,5
Real patient currently on Duvyzat® treatment. Photograph for illustrative purposes only, individual results may vary
*compared to baseline
Duvyzat® (givinostat) is now recommended by NICE
Duvyzat® preserves muscle function*
- Muscle biopsies from Duvyzat-treated patients demonstrated that Duvyzat® prevents muscle deterioration by reducing muscle fibre necrosis, as well as fibrotic and fatty tissue, while increasing muscle tissue 5
- EPIDYS study showed that Duvyzat® reduced muscle fat infiltration across muscle groups required for ambulation (as measured by MRS) 1
Real patient currently on Duvyzat® treatment. Photograph for illustrative purposes only, individual results may vary
*compared to baseline
Duvyzat® delays disease progression*
- EPIDYS study showed that Duvyzat-treated patients performed the four-stair climb (4SC) test significantly faster, preserving motor function longer, compared to the control group at 18 months 1
- EPIDYS study also showed that Duvyzat-treated patients had a lower functional decline with less failed NSAA items than patients in control group 1
- Overall, better outcomes of 4SC and NSAA contribute to a delay in disease progression and lower risk of losing ambulation 6,7
Real patient currently on Duvyzat® treatment. Photograph for illustrative purposes only, individual results may vary
*compared to baseline
A consistent Duvyzat® favourable effect was observed across all EPIDYS efficacy endpoints1
EPIDYS: Primary and key secondary endpoints at week 72 (Group A*)1,8
*All patients were also receiving systemic corticosteroids, in a dose and regimen that was to remain unchanged over the follow-up period.
All key secondary endpoints favoured Duvyzat® vs control, although no statistical significance was reached.
The numbers analysed are 81 for Duvyzat® and 39 for control, except MRS vastus lateralis, which are 77 and 37, respectively. The confidence intervals have not been adjusted for multiplicity and should not be used for hypothesis testing.
4SC: Four-stair Climb, 6MWT: 6-minute Walk Test CI: Confidence Interval, LSM: Least-squares Mean MRS: Magnetic Resonance Spectroscopy NSAA: North Star Ambulatory Assessment TTR: Time-to-rise
Real patient currently on Duvyzat® treatment. Photograph for illustrative purposes only, individual results may vary
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.
- Aartsma-Rus A. Histone deacetylase inhibition with givinostat: a multi-targeted mode of action with the potential to halt the pathological cascade of Duchenne muscular dystrophy. Front Cell Dev Biol. 2025;12:1514898.
- Licandro SA, Crippa L, Pomarico R, et al. The pan HDAC inhibitor Givinostat improves muscle function and histological parameters in two Duchenne muscular dystrophy murine models expressing different haplotypes of the LTBP4 gene. Skelet Muscle. 2021;11(1):19.
- Consalvi S, Mozzetta C, Bettica P, et al. Preclinical studies in the mdx mouse model of duchenne muscular dystrophy with the histone deacetylase inhibitor givinostat. Mol Med. 2013;19(1):79-87.
- Bettica P, Petrini S, D’Oria V, et al. Histological e¦ects of givinostat in boys with Duchenne muscular dystrophy. Neuromuscul Disord. 2016;26(10):643-649.
- Arora H, Willcocks RJ, Lott DJ, et al. Longitudinal timed function tests in Duchenne muscular dystrophy: Imaging DMD cohort natural history. Muscle Nerve. 2018;58(5):631-638.
- Mazzone ES, Pane M, Sormani MP, et al. 24 month longitudinal data in ambulant boys with Duchenne muscular dystrophy. PLoS One. 2013;8(1):e52512.
- Duvyzat Summary of Product Characteristics. EMA, June 2025. https://www.ema.europa.eu/en/documents/product-information/duvyzat-epar-product-information_en.pdf
