Italy-based pharma company Italfarmaco has secured approval from the US Food and Drug Administration (FDA) for its Duchenne muscular dystrophy (DMD) drug Duvyzat (givinostat), following an extended review process. 

The company originally submitted its new drug application in June 2023, with an initial Prescription Drug User Fee Act date of 21 December. The FDA pushed this back to 21 March to have more time to review Italfarmaco’s application. 

DMD is a genetic disorder characterised by progressive muscle degeneration and weakness. It is caused by mutations in the gene that encodes dystrophin, a protein that helps to maintain muscle integrity. Without dystrophin, muscle fibres become damaged and eventually get replaced by fat and connective tissue, leading to muscle weakness and eventual loss of mobility. 

Duvyzat, a histone deacetylase (HDAC) inhibitor, modulates the dysregulated activity of HDACs in the dystrophic muscle, which is a major consequence of the lack of dystrophin—a key factor associated with DMD.  

Following this approval, the drug will enter a busy DMD market, where the top earner, Sarepta’s Elevidys (delandistrogene moxeparvovec), pulled in $200m since it was launched in June 2023. Elevidys is also expected to generate $5.4bn by 2029, according to GlobalData. The gene therapy has been priced at $3.2m per patient, making it one of the world’s most expensive medicines. 

GlobalData is the parent company of Pharmaceutical Technology. 

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The FDA’s decision follows positive results from the Phase III EPIDYS trial (NCT02851797). Data published in The Lancet Neurology journal on 19 March detailed that the trial met its primary endpoint of a change in four-stair climb assessment from the baseline to 72 weeks. This demonstrated that Duvyzat can delay disease progression when added to corticosteroid treatment.  

Key secondary endpoints were also met, including a 40% decline overall in the North Star Ambulatory Assessment (NSAA) total score, and item loss, compared to the control group. Item loss refers to the inability to complete individual tasks in the assessment. There was also a 30% reduction with Duvyzat in the vastus lateralis fat fraction (VLFF), a predictor for the loss of ambulation and disease progression, against the placebo. 

Italfarmaco has established a new US-based subsidiary called ITF Therapeutics to lead the commercial launch of Duvyzat, and to expand the company’s focus on rare diseases, as announced in the 21 March press release. 

In the announcement accompanying the approval, EPIDYS trial investigator Craig Mcdonald said: “Duvyzat’s unique mechanism of action has shown a positive risk/benefit profile and the ability to delay disease progression, supporting its potential to become a key component of the standard of care for people living with DMD.”