Italfarmaco Group Announces New PDUFA Date for Givinostat DMD NDA

Italfarmaco Group announced today that the U.S. Food and Drug Administration (FDA) has extended the review process of the New Drug Application (NDA) for Givinostat, the company’s proprietary histone deacetylase (HDAC) inhibitor for the treatment of Duchenne Muscular Dystrophy (DMD). The new target action date for a decision under the Prescription Drug User Fee Act (PDUFA) is now set for March 21, 2024.

The FDA extended the PDUFA date to allow additional time to review information submitted by Italfarmaco as part of the NDA process. Italfarmaco continues its commitment to work with the FDA to ensure comprehensive support for the NDA evaluation.

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a severe neuromuscular genetic disease characterized by progressive muscle weakness and degeneration and is the most common type of muscular dystrophy globally. DMD is caused by mutations in the DMD gene that result in an absence of a functional dystrophin protein, which plays a crucial role in maintaining the structural and membrane stability of muscle fibers. The disease primarily affects boys with symptoms usually seen between two and five years of age that worsen over time with individuals losing their ability to walk. Eventually, heart and respiratory muscles get affected leading to premature death. DMD incidence is approximately 1 in every 3500 - 6000 male births worldwide.

About Givinostat

Givinostat is an investigational drug discovered through Italfarmaco Group’s internal research and development efforts in collaboration with Lorenzo Puri (Sanford Burnham Prebys Medical Research Institute, San Diego, formerly Santa Lucia Foundation, Rome) and his team, and partnerships with Telethon and Parent Project aps. It is being evaluated for safety and efficacy for the treatment of Duchenne- and Becker- Muscular Dystrophy. Givinostat inhibits histone deacetylases (HDACs). HDACs are enzymes that prevent gene translation by changing the three-dimensional folding of DNA in the cell. Studies show that higher than normal HDAC activity in individuals with DMD may prevent muscle regeneration and also trigger inflammation. Givinostat was observed to slow disease progression, significantly increase muscle mass and reduce the amount of fibrotic tissue and significantly reduce muscle tissue necrosis and fatty replacement (Bettica et al., Neuromuscular Disorders 2016).

About Italfarmaco Group

Italfarmaco Group is a specialty pharmaceutical company engaged in the discovery, development, manufacturing and marketing of branded prescription and nonprescription products in more than 60 countries on 5 continents. Italfarmaco Group’s research and development expertise is best demonstrated through its HDAC inhibitor development programs, addressing new therapeutic treatments of specialty and rare diseases. Through both marketed drugs and compounds in development, Italfarmaco Group is dedicated to serving patients whose needs remain largely unmet.

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