ITF Therapeutics has announced the publication of encouraging long-term data supporting the efficacy and safety of givinostat as a treatment for Duchenne muscular dystrophy (DMD), according to a recent article in The AI Journal. This milestone publication strengthens the case for givinostat, a histone deacetylase (HDAC) inhibitor, as a meaningful therapeutic option for patients living with this devastating genetic disorder.
Duchenne muscular dystrophy is a rare, progressive neuromuscular disease caused by mutations in the dystrophin gene, primarily affecting boys. The condition leads to muscle degeneration, loss of ambulation, and premature death, with few effective long-term treatment options available. Givinostat, developed by ITF Therapeutics, targets the underlying pathophysiology of DMD by inhibiting HDAC, which plays a role in inflammation and fibrotic processes contributing to muscle damage.
The newly published long-term data come from a Phase III clinical study and open-label extension, which evaluated givinostat in ambulant boys with DMD. Over the course of the study, patients receiving givinostat demonstrated significant preservation of muscle function as compared to historical controls. Importantly, the treatment was associated with a slower decline in motor abilities, as measured by standardized tests such as the North Star Ambulatory Assessment (NSAA) and the six-minute walk test (6MWT). These results suggest that givinostat can help maintain mobility and independence in boys suffering from DMD for a greater period than previously possible.
Equally noteworthy, the safety profile of givinostat remained consistent and manageable over the long term. The most common side effects observed were mild to moderate and included gastrointestinal issues, increased creatine phosphokinase (CPK) levels, and temporary changes in blood cell counts. No new or unexpected safety issues emerged with extended use, underscoring givinostat’s potential as a chronic therapy.
The positive findings from the long-term study reinforce earlier research suggesting that HDAC inhibition may offer a disease-modifying approach in DMD. By targeting inflammation and fibrosis, givinostat addresses some of the core drivers of muscle deterioration in DMD, rather than simply managing symptoms. The publication of these robust data supports ITF Therapeutics’ ongoing efforts to bring givinostat to regulatory authorities and, ultimately, to the patient community.
