ITF Therapeutics is the U.S.-based rare disease division of Italfarmaco S.p.A. Founded in 1938 in Milan, Italy, Italfarmaco is a private global pharmaceutical company that has led the successful development of many innovative therapeutic products approved for use by patients around the world. The company operates in more than 60 countries on five continents and continues to advance promising research to address unmet medical needs in a wide range of therapeutic areas.

In January 2024, Italfarmaco launched ITF Therapeutics as a new division in the United States with a focus on the development and commercialization of products to treat rare diseases including DMD. Building on a legacy grounded in collaboration and innovation, ITF Therapeutics strives to partner with leaders from the U.S. patient advocacy and treatment communities to ensure that our programs reflect and support their unique needs and goals. The establishment of ITF Therapeutics also reflects Italfarmaco’s goal to build a world-class team of experts that share a passion to make a positive impact for rare disease communities.

According to the U.S. National Institutes of Health (NIH), there are more than 7,000 known rare diseases that affect approximately 25 to 30 million people in the United States. Currently, there are treatments for only about 600 of these rare diseases. ITF Therapeutics was established with an important goal in mind: to bring new therapeutic options that can address significant unmet needs to people living with rare diseases, and to help make these options available for affected communities in the U.S. Italfarmaco's dedication to rigorous research has fueled our progress in developing our lead product candidate for the treatment of DMD, a rare and severe neuromuscular genetic disease.

DMD is characterized by progressive muscle weakness and degeneration and is the most common type of muscular dystrophy globally. It is caused by mutations in the DMD gene leading to alterations in dystrophin expression and dysregulation of muscle repair. Impairment of muscle repair typically leads to chronic inflammation, muscle degeneration and eventual replacement of muscle tissue with scar tissue and fatty tissue. Without dystrophin, muscle fibers are highly susceptible to injury, leading to chronic inflammations, impairment of muscle regeneration and muscle tissue replacement by fibrotic and fatty tissue. DMD primarily affects boys. Symptoms usually appear between 2 and 5 years of age and worsen over time, eventually impairing the ability to walk and diminishing other functions reliant on healthy muscle. DMD incidence is approximately 1 in every 3,500-6,000 male births worldwide.

Based on detailed understanding of disease onset and progression, Italfarmaco led the early-stage research and clinical development of DUVYZAT^™ (givinostat), a histone deacetylase (HDAC) inhibitor, as a potential treatment for DMD. The development of DUVYZAT was made possible with support from members of the DMD community whose valued contributions played a critical role at every stage of the process.

The regulatory review and approval of DUVYZAT in the U.S. was based on Italfarmaco’s multi-year clinical development program, including the pivotal Phase 3 EPIDYS clinical trial in patients with DMD. DUVYZAT was approved by the U.S. Food and Drug Administration (FDA) on March 21, 2024 as a treatment for patients 6 years of age and older with DMD and is currently under review by the European Medicines Agency as a potential treatment for DMD. The therapy is also in early-stage development as a potential treatment for Becker muscular dystrophy.

For additional information about DUVYZAT, read our press release.

ITF Therapeutics is privileged to collaborate with advocacy organizations that provide essential support to patients and caregivers. For information about our engagement with patient advocacy organizations, please contact Caroline Allen, US Patient Advocacy and Communications Lead, at c.allen@itftherapeutics.com.

Learn more about organizations dedicated to bringing information and support services to individuals and families affected by DMD: