Italfarmaco Updates on Givinostat for Duchenne Muscular Dystrophy
Italfarmaco S.p.A., a global pharmaceutical company dedicated to developing innovative treatments for rare diseases, has provided a comprehensive update on the regulatory and clinical advancements of givinostat, its investigational therapy for Duchenne muscular dystrophy (DMD). The company outlined key regulatory milestones, progress in clinical trials, and its broader commitment to ensuring global access to the treatment for patients affected by DMD.
Regulatory Milestones and Global Expansion
Givinostat has made significant regulatory progress, achieving important approvals that mark a crucial step forward in making this therapy available to patients worldwide. In March 2024, the U.S. Food and Drug Administration (FDA) granted full approval for givinostat for patients six years and older, regardless of their ambulatory status. This approval underscores the potential of givinostat as a treatment option for a broad spectrum of DMD patients.
In December 2024, the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) approved givinostat for ambulant patients aged six years and older. Additionally, the agency granted conditional approval for non-ambulant patients, allowing for expanded access to the therapy while further clinical data are collected.
Beyond the U.S. and UK, Italfarmaco is actively pursuing regulatory approvals in other regions. The European Medicines Agency (EMA) is currently reviewing the Marketing Authorisation Application (MAA) for givinostat. A decision from the Committee for Medicinal Products for Human Use (CHMP) is expected in the first half of 2025. Meanwhile, the company is in discussions with regulatory agencies in additional countries to further expand access to givinostat, ensuring that more patients worldwide can benefit from this potentially life-changing treatment.
Carlos Barallobre, CEO of Italfarmaco, emphasized the company’s unwavering dedication to the DMD community, stating, “At Italfarmaco, our mission is clear: to bring innovative and effective therapies to patients with Duchenne muscular dystrophy. The approvals of givinostat in the U.S. and UK represent major milestones in our journey, but our work is far from over. We remain committed to accelerating the regulatory process worldwide so that every patient, regardless of location, has timely access to a treatment that can alter the course of this devastating disease.”
Ongoing Clinical Trials: Advancing Research and Expanding Indications
To further evaluate the long-term safety and efficacy of givinostat and expand its potential indications, Italfarmaco is conducting multiple clinical trials across various patient populations. These trials are designed to provide additional evidence supporting the drug’s effectiveness and its potential role in improving disease outcomes for a wider range of DMD patients.
Currently, three major clinical trials are underway in Europe and Canada:
- NCT05933057: This study focuses on non-ambulant patients aged nine years and older. By evaluating the efficacy and safety of givinostat in this population, the trial aims to provide critical insights into its benefits for patients in the later stages of DMD progression.
- NCT06769633: This trial is assessing the pharmacokinetics and safety of givinostat in younger patients between two and five years of age. Recruitment has recently been completed for the first cohort of four- to five-year-olds, marking a significant step in understanding how the drug performs in early-stage DMD patients.
- NCT03373968: A long-term follow-up study designed to confirm the tolerability and sustained efficacy of givinostat, this trial includes patients who have been receiving the drug for up to eleven years. The findings from this study will provide valuable data on the prolonged impact of givinostat therapy in individuals with DMD.
Dr. Paolo Bettica, MD, PhD, Chief Medical Officer at Italfarmaco Group, highlighted the significance of these ongoing trials, stating, “Our research efforts are focused on further validating givinostat’s clinical benefits and reinforcing its role as a crucial treatment option for Duchenne muscular dystrophy. The limited number of effective treatments currently available underscores the need for continued innovation in this space. By targeting the biological mechanisms responsible for muscle degeneration, givinostat holds the potential to change the trajectory of this disease.”
The Science Behind Givinostat: A Targeted Approach to DMD Treatment
Duchenne muscular dystrophy is a genetic disorder caused by mutations in the DMD gene, leading to the absence of dystrophin, a crucial protein responsible for muscle cell stability and repair. The loss of dystrophin results in progressive muscle deterioration, chronic inflammation, and the replacement of muscle tissue with fat and fibrotic tissue. Over time, DMD leads to significant mobility loss, respiratory complications, and, ultimately, life-threatening cardiac issues.
Givinostat is an oral histone deacetylase (HDAC) inhibitor that targets key biological pathways involved in DMD progression. By modulating gene expression and reducing inflammation, givinostat aims to preserve muscle tissue, slow disease progression, and enhance muscle function. Unlike conventional therapies that focus solely on replacing dystrophin, givinostat takes a broader approach by addressing the downstream effects of dystrophin deficiency, making it a promising option for patients at various stages of the disease.
Global Impact and Commitment to the DMD Community
Duchenne muscular dystrophy primarily affects boys, with symptoms typically emerging between the ages of two and five. The disease is characterized by progressive muscle weakness, loss of mobility, and life-threatening complications affecting the heart and respiratory muscles. With a worldwide incidence of approximately 1 in 5,050 male births, DMD remains one of the most severe and common forms of childhood muscular dystrophy.
Given the urgent need for effective treatments, Italfarmaco is working closely with healthcare providers, patient advocacy groups, and regulatory agencies to ensure that givinostat reaches as many patients as possible. The company is also actively involved in initiatives aimed at improving disease awareness, supporting early diagnosis, and expanding access to care.
As regulatory reviews continue and clinical trials progress, Italfarmaco remains dedicated to advancing DMD research and improving patient outcomes. With givinostat’s approvals in the U.S. and UK, and ongoing efforts to secure additional regulatory clearances, the company is making significant strides toward addressing the unmet medical needs of the global DMD community.
Carlos Barallobre reinforced this commitment, stating, “For families affected by Duchenne muscular dystrophy, time is of the essence. We are accelerating our efforts on all fronts—research, regulatory approvals, and global accessibility—to ensure that patients can benefit from givinostat as soon as possible. We are deeply grateful to the clinicians, researchers, and, most importantly, the patients and families who continue to support our mission.”
The Future of Givinostat in DMD Treatment
With a strong foundation of clinical data, ongoing regulatory advancements, and an expanding global footprint, givinostat is positioned to become a cornerstone therapy in the management of Duchenne muscular dystrophy. As Italfarmaco continues to drive research and development, the company remains steadfast in its goal of transforming the standard of care for individuals living with DMD.
Through continued collaboration with the medical community and patient organizations, Italfarmaco is not only advancing a promising therapy but also contributing to a future where children and young adults with DMD have access to better treatment options, improved quality of life, and renewed hope.
About Givinostat
Givinostat was discovered through Italfarmaco’s research and development efforts in collaboration with Telethon and Duchenne Parent Project (Italy). Givinostat is an orally administered histone deacetylases (HDAC) inhibitor. HDAC is activity upregulated in DMD muscle and has the potential to modify the expression of certain genes and biological processes involved in muscle repair and inflammation.10, 11
About ITALFARMACO
Founded in 1938 in Milan, Italy, Italfarmaco is a private global pharmaceutical company that has led the successful development and approval of many pharmaceutical products around the world. The Italfarmaco group has operations in more than 60 countries through directly controlled or affiliated companies. The company is a leader in pharmaceutical research, product development, production and commercialisation with proven success in many therapeutic areas including immuno-oncology, gynaecology, neurology, cardiovascular disease and rare diseases. Italfarmaco’s rare disease unit includes programmes in Duchenne muscular dystrophy, Becker muscular dystrophy, amyotrophic lateral sclerosis and polycythaemia vera.
