Duchenne muscular dystrophy: Prix Galien awarded to Italian drug that slows the progression of the disease

Givinostat has won the Prix Galien Italia in the ‘Rare Diseases’ category; this molecule ‘has been shown in clinical trials to slow the progression of Duchenne muscular dystrophy’ thanks to a new mechanism of action. The drug represents a story of Italian scientific excellence and is the result of research and development by Italfarmaco, a private global pharmaceutical company founded in 1938 in Milan.

The award ceremony took place in Turin. “Winning the Prix Galien recognises not only an innovative molecule such as givinostat, but also an entrepreneurial and scientific journey built up over time through constant investment in research and development,” explains Francesco Di Marco, Group CEO. The award takes on even greater significance because it comes in a field characterised by significant unmet medical needs and a pressing need for innovation, where treatment options have long been limited. This achievement highlights the path of innovation undertaken by Italfarmaco and the capacity of Italian research to generate scientific progress and translate it into concrete therapeutic solutions for patients.”
“This prestigious award fills us with pride,” says Julie De Santis, a member of the Board of Directors of Italfarmaco Holding. “Italfarmaco was founded in 1938 in Milan. Building on our family tradition, we are guided by a long-term vision, deeply rooted in our heritage and supported by an international team. Over the past 88 years, the company has consistently invested in research and development, and givinostat is the result of this commitment. We wish to share the Prix Galien with the clinicians and patient organisations that have made a decisive contribution to the development of the molecule. Our aim is to provide solutions to unmet clinical needs, particularly in the field of rare diseases, and thanks to givinostat, a significant step forward has been taken.”

“The result of Italian research, givinostat – as stated in the citation for the award – represents a therapeutic innovation in Duchenne muscular dystrophy because it introduces a mechanism of action that is distinct from and independent of the specific genetic mutation, with the potential to be applied to a broad population of patients eligible under the authorised indications. When used in combination with corticosteroids, it has been shown to slow the progression of the disease and to help maintain motor function and independence for longer, addressing a significant therapeutic need in the field of rare diseases, with a significant impact on the quality of life of patients and their families.”

Duchenne muscular dystrophy is a rare genetic neuromuscular disorder that affects almost exclusively boys and is caused by the absence or severe deficiency of dystrophin. When this protein is absent or does not function properly, the muscle fibres become more fragile and, over time, an inflammatory process is triggered, leading to the replacement of muscle tissue with fibrous tissue.

“As the disease progresses, it leads over time to a significant loss of muscle strength, mobility and independence,” emphasises Eugenio Maria Mercuri, Full Professor at the Catholic University of the Sacred Heart. Patients also face serious complications such as cardiomyopathies, respiratory and neurological problems, with a significant impact on cognitive function. Givinostat, which acts on the cellular mechanisms contributing to muscle damage, is a major step forward in treatment, as it significantly slows the progression of the disease. The drug belongs to the class of histone deacetylase inhibitors, proteins that regulate the activity of numerous genes. One of the molecule’s key features is its ability to offer a therapeutic option regardless of the specific genetic mutation, thereby broadening the number of patients who can benefit from the treatment and helping to preserve patients’ motor function and independence over time.”