Positive Phase III Outcome for Italfarmaco's Duchenne Muscular Dystrophy Drug

Italian pharmaceutical firm Italfarmaco has announced the complete findings from its Phase III clinical trial of givinostat, a drug intended for patients suffering from Duchenne muscular dystrophy (DMD). The trial, known as Epidys, successfully achieved its primary goal, demonstrating that givinostat can effectively slow the progression of the disease when combined with corticosteroid therapy.

The study, published in The Lancet Neurology, focused on the change in the four-stair climb assessment from the beginning of the study to 72 weeks. The drug's efficacy was evident in the slower decline in patients' ability to perform this functional task compared to those given a placebo, with a significant difference of 1.78 seconds and a p-value of 0.037.

The trial included 179 male patients with DMD, aged six and above, from North America and Europe. Italfarmaco highlighted that the drug not only delayed the disease's progression but also showed positive results in secondary endpoints related to muscle function and strength. Givinostat was linked to a 40% reduction in the decline of the NSAA total score and a 30% decrease in the vastus lateralis fat fraction (VLFF), which is an indicator of ambulation loss and disease progression.

The most frequently reported adverse events related to the drug were a decrease in platelet count, hypertriglyceridemia, abdominal pain, and diarrhea. However, no patients withdrew from the study due to these side effects.

The FDA is expected to make a decision on the approval of givinostat on March 21. The drug is an oral medication designed to inhibit histone deacetylases (HDACs), enzymes that play a crucial role in muscle gene expression. Italfarmaco initially submitted its New Drug Application (NDA) for givinostat in June 2023, following the release of preliminary data in 2022.

In the DMD treatment market, Sarepta's drug Elevidys has been notably successful, generating over $200 million in revenue since its launch in the latter half of the previous year.

The success of Italfarmaco's givinostat could have a significant impact on the DMD treatment landscape, offering a new therapeutic option for patients and potentially contributing to the company's growth and reputation in the pharmaceutical industry.