Italfarmaco Group announced today that its Marketing Authorization Application (MAA) for Givinostat as a potential treatment for Duchenne Muscular Dystrophy (DMD) has been submitted to the European Medicine Agency (EMA) and that the EMA has started its regulatory review process.

The MAA submission was based on the safety and efficacy results from the Phase 3 EPIDYS clinical trial which tested Givinostat, a histone deacetylase (HDAC) inhibitor, in patients with DMD. The MAA submission to the EMA follows Italfarmaco Group's New Drug Application (NDA) submission for Givinostat to the U.S. Food and Drug Administration (FDA), which has granted priority review for Givinostat as announced in June this year.

“The Phase 3 evaluation of Givinostat demonstrated that it can slow DMD progression in boys six years and older. If approved, Givinostat could greatly benefit younger and older DMD patients by preserving their motor skills and muscle strength,” said Paolo Bettica, MD, PhD, Chief Medical Officer at Italfarmaco Group. “Now that our market authorization application is under review, we are looking forward to working with the regulatory authorities to bring this treatment option to patients affected by this devastating disease with the goal of providing therapeutic benefit and increasing their quality of life.”

“The acceptance of our submissions to European and U.S. regulatory bodies is a tremendous achievement for the company and reflects Italfarmaco’s research efforts and commitment to the successful clinical development of Givinostat,” added Carlos Barallobre, CEO at Italfarmaco Group.

The pivotal Phase 3 clinical trial EPIDYS evaluated the efficacy and safety of Givinostat in 179 ambulant DMD patients six years and older (Clinicaltrials.gov: NCT02851797). Givinostat was administered in oral formulation for 18 months, along with continued corticosteroid treatment. The study met its primary endpoint, assessing functional improvement through mean change from baseline in time to climb four stairs in the target population.¹ Analysis of key secondary endpoints, encompassing fat infiltration evaluation by magnetic resonance, muscle function and strength tests, yielded results consistent with the primary endpoint. The safety and tolerability profile of Givinostat was notably in accordance with previous study results.

In DMD patients, the absence of the dystrophin protein detrimentally alters various biological processes within muscle tissues, resulting in progressive muscle degeneration and eventually, premature death. The deregulated activity of HDACs, a group of enzymes that contribute to the maintenance of skeletal muscle homeostasis, is a major adverse consequence of the lack of dystrophin. By inhibiting HDAC pathological overactivity, Givinostat is able to remedy the aberrant cascade of intracellular signaling pathways leading to muscle damage, counteracting the disease pathology and slowing down muscle deterioration.

About Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy (DMD) is a severe neuromuscular disease characterized by progressive muscle weakness and degeneration and is the most common type of muscular dystrophy globally. 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 was observed to slow disease progression by reducing inflammation, intramuscular fibrosis, muscle tissue necrosis and fatty replacement while significantly increasing muscle mass (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 acceptance of our submissions to European and U.S. regulatory bodies is a tremendous achievement for the company and reflects Italfarmaco’s research efforts and commitment to the successful clinical development of Givinostat.