Italfarmaco Presents New Cardiac Data for Givinostat at 16th European Paediatric Neurology Society (EPNS) Congress

GlobeNewswire | Italfarmaco
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  • In total, 11 abstracts on givinostat were accepted for presentation at the Congress, including two for an oral presentation  

MILAN, Italy, July 11, 2025 – Italfarmaco S.p.A. announced today that new cardiac data for givinostat, a novel histone deacetylase (HDAC) inhibitor, in ambulant boys with Duchenne muscular dystrophy (DMD) will be presented at the European Paediatric Neurology Society (EPNS) Congress, taking place July 8 - 12, 2025 in Munich, Germany. Post hoc analysis from the completed Phase 3 EPIDYS trial showed less decline in cardiac function throughout the study, compared to those who received placebo. In addition, the analysis showed no prolongation of the QTc interval in either the placebo or givinostat treatment groups. In a separate crossover study conducted by Italfarmaco in healthy volunteers to assess the impact of a therapeutic and supratherapeutic dose of givinostat, the administration of the therapeutic dose was not associated with any risk of QTc interval prolongation.

“Individuals with Duchenne are at risk of cardiac complications as the disease progresses. The beneficial trend observed with givinostat in preserving heart function compared to placebo is encouraging,” stated Paolo Bettica, MD, PhD, Chief Medical Officer at Italfarmaco Group. “Furthermore, these data support the cardiac safety profile of givinostat, providing additional confidence for its use. We are pleased to share these findings at the EPNS Congress.”

Individuals with DMD experience progressive skeletal muscle degeneration, and over time, they are also at risk of developing impaired cardiac function. Reduced mobility and the underlying genetic defect contribute to an increased risk of cardiomyopathy and arrhythmias, making routine cardiac monitoring a critical component of clinical care and disease management. In the EPIDYS study, 179 ambulatory boys aged 6 years and older with genetically confirmed DMD were assessed throughout the 72-week trial on electrocardiogram (ECG) and echocardiogram (ECHO) parameters. At the end of the study, the givinostat-treated group showed a beneficial trend compared to the placebo group.

In a separate Phase 1 crossover study, Italfarmaco evaluated the cardiac safety profile of a therapeutic and supratherapeutic dose of givinostat in healthy volunteers. The data demonstrated that the therapeutic dose of givinostat is not expected to pose a risk of QTc prolongation. The therapeutic dose of givinostat had no clinically relevant effect on heart rate or cardiac conduction, nor did it have a clinically relevant effect on the QTc interval.

A total of 11 abstracts related to givinostat were accepted for either oral or poster presentation at the EPNS Congress. The list of all abstracts accepted for presentations can be accessed on the EPNS website using the search term “givinostat.”

The European Commission (EC) granted givinostat (Duvyzat®) conditional marketing authorisation in the EU in June 2025 to ambulant DMD patients 6 years and older. Givinostat was also granted approval by the US Food and Drug Administration (FDA) in March 2024 for the treatment of DMD patients 6 years and older. In December 2024, the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) approved givinostat for patients 6 years and older who are ambulatory and granted conditional marketing approval for non-ambulatory patients.

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a rare, progressive neuromuscular disorder caused by mutations in the DMD gene. Mutations in the DMD gene prevent the production of functional dystrophin, causing the dystrophin-associated protein complex (DAPC) to break down. This makes muscle fibres more vulnerable to damage and increases histone deacetylase (HDAC) levels in the muscle cells, blocking the activation of important genes needed for muscle maintenance and repair. As a result, muscle fibres experience ongoing damage, leading to chronic inflammation and poor regeneration. Over time, muscle cells die and are replaced by scar tissue and fat.1-4 DMD primarily affects males, with symptoms typically appearing between the ages of two and five. As the condition progresses, muscle weakness worsens, leading to difficulty walking and eventually to loss of ambulation. Over time, the heart and respiratory muscles are also affected, which are the leading causes of premature death.5 DMD is one of the most severe and common forms of childhood muscular dystrophy, with a global birth incidence of approximately 1 in 5,050 boys.6

About ITALFARMACO

Founded in 1938 in Milan, Italy, Italfarmaco is a private global pharmaceutical company that has led the successful development and approval of many pharmaceutical products around the world. The Italfarmaco group has operations in more than 90 countries through directly controlled or affiliated companies. The company is a leader in pharmaceutical research, product development, production and commercialisation with proven success in many therapeutic areas including immuno-oncology, gynaecology, neurology, cardiovascular disease and rare diseases. Italfarmaco's rare disease unit includes programmes in Duchenne muscular dystrophy, Becker muscular dystrophy, amyotrophic lateral sclerosis and polycythaemia vera.


Media enquiries:
Anja Heuer / Adolfo Luna | +49 (0) 151 106 199 05 | italfarmaco@trophic.eu

Other enquiries:
Samantha Parker | Patient Advocacy and Communications Lead | RDEnquiries@italfarmacogroup.com


References:

  1. Sandonà M, Cavioli G, Renzini A, et al. Histone Deacetylases: Molecular Mechanisms and Therapeutic Implications for Muscular Dystrophies. Int J Mol Sci. 2023;24(5):4306. https://doi.org/10.3390/ijms24054306.
  2. Consalvi S, Saccone V, Giordani L, Minetti G, Mozzetta C, Puri PL. Histone Deacetylase Inhibitors in the Treatment of Muscular Dystrophies: Epigenetic Drugs for Genetic Diseases. Mol Med. 2011;17(5):457–465. https://doi.org/10.2119/molmed.2011.00049.
  3. Bez Batti Angulski A, Hosny N, Cohen H, et al. Duchenne muscular dystrophy: disease mechanism and therapeutic strategies. Front Physiol. 2023;14:1183101. https://doi.org/10.3389/fphys.2023.1183101.
  4. Giuliani G, Rosina M, Reggio A. Signaling pathways regulating the fate of fibro/adipogenic progenitors (FAPs) in skeletal muscle regeneration and disease. FEBS J. 2022;289(21):6484–6517. https://doi.org/10.1111/febs.16080.
  5. Walter MC, Reilich P. Recent developments in Duchenne muscular dystrophy: facts and numbers. J Cachexia Sarcopenia Muscle. 2017;8(5):681–685. https://doi.org/10.1002/jcsm.12245.
  6. Crisafulli S, Sultana J, Fontana A, Salvo F, Messina S, Trifirò G. Global epidemiology of Duchenne muscular dystrophy: an updated systematic review and meta-analysis. Orphanet J Rare Dis. 2020;15(1):141. https://doi.org/10.1186/s13023-020-01430-8.

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