Italfarmaco Updates on Givinostat for Duchenne Muscular Dystrophy: Access, Regulations, and Trials
Italfarmaco S.p.A. has announced significant progress in the regulatory and clinical development of givinostat, its investigational drug for the treatment of Duchenne muscular dystrophy (DMD). The company provided a comprehensive update on key regulatory approvals, ongoing clinical trials, and its commitment to increasing global access to the therapy.
Regulatory Milestones
Givinostat has recently achieved critical regulatory milestones in both the United States and the United Kingdom, further advancing its availability to patients with DMD. In March 2024, the U.S. Food and Drug Administration (FDA) granted full approval for givinostat for ambulant patients aged six years and older. In the UK, givinostat received full approval for ambulant patients aged six years and older, while conditional approval was granted for non-ambulant patients, highlighting the drug’s potential to address a wide range of DMD patients.
Italfarmaco is actively working on regulatory filings in additional countries to expand global access to givinostat. The European Medicines Agency (EMA) is currently reviewing the company’s Marketing Authorisation Application (MAA) for givinostat, with the Committee for Medicinal Products for Human Use (CHMP) opinion expected in the first half of 2025. These efforts reflect Italfarmaco’s commitment to ensuring that patients across the world have access to effective therapies for DMD.
Carlos Barallobre, CEO of Italfarmaco, emphasized the company’s dedication to the DMD community: “At Italfarmaco, our unwavering commitment is to the Duchenne community. The givinostat approvals in the US and UK, along with the promising long-term data we are generating, bring us closer to our goal: ensuring that every patient, regardless of location, has access to a therapy that can make a difference in disease progression. We are diligently working with global regulatory bodies to expedite availability, because we understand that for patients and families affected by DMD, every moment counts.”
Ongoing Clinical Trials in DMD
Italfarmaco is also conducting a series of ongoing clinical trials aimed at expanding the potential use of givinostat and providing long-term follow-up data on patients treated with the drug. These trials, which are taking place in Europe and Canada, include:
- NCT05933057: This trial is evaluating the safety and efficacy of givinostat in non-ambulant patients aged 9 years and older.
- NCT06769633: This study is focused on the pharmacokinetics and safety of givinostat in younger patients aged 2 to 5 years. Recruitment for the first cohort of 4- to 5-year-olds has recently been completed.
- NCT03373968: This trial is aimed at confirming the long-term tolerability and efficacy of givinostat, with follow-up data being collected for up to eleven years.
Paolo Bettica, MD, PhD, Chief Medical Officer at Italfarmaco Group, highlighted the importance of these clinical trials: “Our continuing clinical trial evaluation seeks to further affirm givinostat’s clinical value in Duchenne muscular dystrophy, where treatment options remain limited, and by precisely targeting the biological drivers of muscle degeneration. We look forward to our ongoing interactions with the regulatory bodies and thank the clinicians for their tireless efforts and the DMD community and families for their continuing trust.”
Understanding Duchenne Muscular Dystrophy (DMD)
Duchenne muscular dystrophy (DMD) is a progressive neuromuscular disorder caused by a mutation in the DMD gene, which leads to the absence of dystrophin, a protein essential for maintaining muscle cell integrity. The absence of dystrophin makes muscle fibers more vulnerable to damage, leading to chronic inflammation, muscle degeneration, and eventual replacement of muscle tissue by fat and fibrotic tissue. The disease predominantly affects boys, with symptoms typically appearing between the ages of two and five. As the disease progresses, it impairs the ability to walk and eventually affects the heart and respiratory muscles, which are the leading causes of premature death. DMD is one of the most severe forms of childhood muscular dystrophy, with a global birth incidence of about 1 in 5,050 boys.
About Givinostat
Givinostat, discovered through Italfarmaco’s research and development efforts in collaboration with Telethon and Duchenne Parent Project (Italy), is an orally administered histone deacetylase (HDAC) inhibitor. HDAC activity is upregulated in DMD muscle, and givinostat has the potential to modify the expression of specific genes and biological processes involved in muscle repair and inflammation. By targeting the biological mechanisms driving muscle degeneration in DMD, givinostat aims to slow disease progression and improve the quality of life for patients.
