-- Submission based on completed Phase 3 program in 179 boys demonstrating significant slowing of disease progression with a good safety and tolerability profile --

-- PDUFA goal date set for December 21, 2023 --

Italfarmaco Group announced today that the U.S. Food and Drug Administration (FDA) has completed its filing review and accepted the company’s New Drug Application (NDA) for Givinostat. Givinostat is the company’s proprietary histone deacetylase (HDAC) inhibitor for the treatment of Duchenne Muscular Dystrophy (DMD). The compound was granted priority review by the FDA with a Prescription Drug User Fee Act (PDUFA) goal date set for December 21, 2023.

“The FDA’s communication on our NDA and the priority review for Givinostat represents a major milestone for Italfarmaco and is a significant step forward in the search for effective treatments against this debilitating rare disease. In our Phase 3 trial, Givinostat demonstrated that it can slow DMD progression, thereby potentially giving young boys and their families a treatment option to retain motor function,” said Paolo Bettica, MD, PhD, Chief Medical Officer at Italfarmaco Group. “We are excited to be a part of the increasing momentum in this area of medicine with new treatments reaching the market and we hope to achieve a positive impact on the lives of younger and older DMD patients, their families and the medical community that serves them.”

“This significant accomplishment stands as the best evidence of Italfarmaco's dedication to R&D and the robust clinical development strategy we have followed,” said Carlos Barallobre, CEO at Italfarmaco Group. “We are also in the process of submitting an application for market authorization in Europe to facilitate the reach of these advancements to those in need as swiftly and efficiently as possible.”

The NDA submission is grounded in Italfarmaco Group’s completed clinical development program including the pivotal Phase 3 clinical trial EPIDYS which demonstrated positive results assessing the efficacy and safety of Givinostat in patients with DMD (Clinicaltrials.gov: NCT02851797). The study included 179 ambulant boys 6 years and older, who continued steroid treatment with the addition of an oral suspension of Givinostat for 18 months. The primary endpoint evaluated functional improvement through mean change from baseline in time to climb four stairs in the target population¹. Key secondary endpoints included various muscle function and strength tests as well as fat infiltration assessment by magnetic resonance. The study met the primary endpoint with consistent results in the key secondary endpoints. Givinostat continued to demonstrate a tolerability profile in line with previous studies.

Dr. Bettica will discuss Givinostat’s mode of action and the clinical results to date at the Parent Project Muscular Dystrophy 2023 annual conference on Friday, June 30, 2023, in the “Research Row: Approved or on the Horizon” session.

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.

¹ Target population: Individuals with a baseline vastus lateralis muscle fat fraction (VL MFF) assessed by magnetic resonance spectroscopy (MRS) in the range >5% and ≤ 30%

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The FDA’s communication on our NDA and the priority review for Givinostat represents a major milestone for Italfarmaco and is a significant step forward in the search for effective treatments against this debilitating rare disease.