ITF Therapeutics LLC is dedicated to bringing new therapeutic options to people living with rare diseases.

Who We Are

In all areas of our work, ITF Therapeutics is guided by the voices of the communities we serve. We value the special role that personal stories and experiences play in helping us understand the impact that rare diseases can have on patients and caregivers. The voice of the patient community also helps us understand the most effective ways to partner with and support patients, families, and healthcare professionals. It is an honor for us to collaborate with leaders in advocacy, research, and patient care as we work to build a brighter future for people living with rare diseases such as Duchenne muscular dystrophy (DMD).

About ITF Therapeutics

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 90 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.


Working in close collaboration with patient advocates and clinical leaders, our goal is to help make a positive difference for people living with many different rare diseases.

Matt Trudeau, General Manager, ITF Therapeutics

Our Focus on Rare Diseases

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 may 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 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.

ITF Therapeutics builds on Itafarmaco’s long history of breaking barriers in research and innovation to bring new therapeutic options to patients, and we now can apply that experience to address the needs of individuals and families affected by rare diseases including DMD.

Carlos Barallobre, CEO, Italfarmaco


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 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.

Please see Important Safety Information and Medication Guide.

DOWNLOAD press release

Expanded Access Policy
ITF Therapeutics does not have an investigational product candidate available for expanded access at this time in the US. We believe that participation in clinical trials, which are carefully designed to determine the safety and efficacy of an investigational therapy, is the most appropriate way for patients to access an investigational therapy until sufficient evidence is available that suggests the potential benefit outweighs the risk.

Our Product

Duvyzat Logo

Givinostat is marketed in the U.S. under the brand name DUVYZAT (givinostat). Learn more by visiting


ITF Therapeutics Leadership

The ITF Therapeutics team includes experts in all areas of rare disease product development and commercialization who share a passion to make a positive difference for people living with DMD and other rare diseases.

Patient Advocacy

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

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

Cure Duchenne Logo
Jett Foundation Logo
Little Hercules Foundation Logo
Muscular Dystrophy Association
Parent Project
Team Joseph
The Akari Foundation

Additional resources for the Rare Disease Community

National Organization for Rare Disorders
Every Life Foundation Logo
Global Genes Logo

Updates and additional information related to DUVYZAT will be shared via

Company News


Learn more about our research in rare diseases including DMD.

Muscular Dystrophy Association Clinical & Scientific Conference 2024

Givinostat study in DMD: supportive results

Givinostat effects on DMD pathogenesis


ITF Therapeutics is developing our US team into a world-class group of experts that share a passion to make a positive impact for rare disease communities.

If you have a collaborative, innovative spirit and are looking to be part of a growing team from the ground up, we'd love to hear from you.

Check out our LinkedIn page for a listing of some of our current open positions.

Job Postings

Contact Us

ITF Therapeutics LLC
300 Baker Avenue, Suite 280
Concord, MA 01742

For medical inquiries from healthcare providers, please contact:

For all other inquiries, including general inquiries from patients and caregivers, please contact: