FDA accepts NDA for givinostat to treat Duchenne muscular dystrophy


According to Italfarmaco Group, a New Drug Application (NDA) for givinostat has been accepted and granted priority review by the FDA to treat Duchenne muscular dystrophy (DMD) patients, following positive topline phase 3 trial results.

FDA accepts NDA for givinostat to treat Duchenne muscular dystrophy | Image Credit: © luchschenF - © luchschenF - stock.adobe.com.

FDA accepts NDA for givinostat to treat Duchenne muscular dystrophy | Image Credit: © luchschenF - © luchschenF - stock.adobe.com.

The FDA has accepted the New Drug Application (NDA) for givinostat (ITF2357; Italfarmaco Group), a proprietary histone deacetylase (HDAC) inhibitor to treat Duchenne muscular dystrophy (DMD), according to a press release from Italfarmaco Group. Givinostat received priority review from the FDA and has a Prescription Drug User Fee Act (PDUFA) date of December 21, 2023.

The NDA acceptance follows positive results from the pivotal phase 3 EPIDYS (NCT02851797) clinical trial, evaluating the safety and efficacy of givinostat in DMD patients, in which 179 boys aged 6 years and older participated. Patients continued steroid treatment while adding an oral suspension of givinostat for 18 months. Functional improvement in the target population via mean change from baseline in time to climb four stairs was the primary endpoint. Muscle function and strength tests and fat infiltration assessment by magnetic resonance were key secondary outcomes. The target population included individuals with a baseline vastus lateralis muscle fat fraction (VL MFF) in the range > 5% and ≤ 30%, assessed by magnetic resonance spectroscopy (MRS). Results demonstrated that the primary outcome was met while consistent results were reported for secondary outcomes, maintaining a tolerability profile consistent with previous studies.

DMD primarily impacts boys, who see symptoms between ages 2 and 5 years that worsen over time, “with individuals losing their ability to walk.” Heart and respiratory muscles are eventually affected, which can lead to premature death. Worldwide, the rare disease occurs in approximately 1 in every 3500 to 6000 males.

Higher HDAC, enzymes that change the 3-dimensional folding of DNA in a cell to prevent gene translation, activity in DMD patients can prevent muscle regeneration and trigger inflammation. According to the press release, givinostat was observed to slow progression of DMD, significantly increase muscle mass and reduce fibrotic tissue, and significantly reduce muscle tissue necrosis and fatty replacement.

The FDA-accepted NDA of givinostat comes 5 days after the FDA approved delandistrogene moxeparvovec-rokl (ELEVIDYS; Sarepta Therapeutics, Inc) to treat ambulatory pediatric DMD patients aged 4 to 5 years, making it the first gene therapy to treat the indicated patient population.


Italfarmaco Group completes FDA submission of New Drug Application for givinostat in Duchenne muscular dystrophy and receives priority review. Businesswire. June 29, 2023. Accessed June 29, 2023. https://www.businesswire.com/news/home/20230629900551/en/Italfarmaco-Group-Completes-FDA-Submission-of-New-Drug-Application-for-Givinostat-in-Duchenne-Muscular-Dystrophy-and-Receives-Priority-Review

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