Italfarmaco

This is a randomised, double-blind, placebo-controlled, multicentre study to evaluate the efficacy, safety, and tolerability of givinostat in non-ambulant male paediatric (aged 9 to \<18 years) patients with DMD. 138 patients will be randomised 2:1 to givinostat or placebo and will be treated for 18 months. * Planned screening duration: approximately 4 weeks (±14 days) * Planned treatment duration: 18 months (approximately 72 weeks) * Planned follow-up duration: 4 weeks (±7 days) (for patients not participating in the long-term safety study) * Total duration of study participation: up to 83 weeks (ie, 20-21 months)Show more

This is a prospective observational study conducted to evaluate safety, tolerability, and functional outcomes of patients with DMD newly initiating oral givinostat or having started therapy within 6 months as part of routine clinical care in the US. The study has a planned maximum duration of 5 years for the first enrolled patients, including a 24-month enrollment period and a minimum of 2 years of follow-up.Show more

The goal of this clinical trial is to compare the efficacy and safety of givinostat to hydroxyurea in Jak2V617F-positive high risk polycythemia vera patients.

This is an open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD (Duchenne's muscular dystrophy) patients who have been previously treated in one of the GIVINOSTAT studies.

This is a Phase 2 Open-label (Core Phase Plus Extension Phase) With 2 Cohorts Study to Assess the Pharmacokinetics and Safety of Givinostat in younger DMD Patients. * Planned screening duration: approximately 4 weeks * Planned Core Treatment duration: approximately 48 weeks * Planned Extension Treatment duration: approximately 96 weeks * Planned Follow Up duration: approximately 4 weeks (± 7 days) * Total duration of study participation: up to 151 weeks (ie, 37-38 months)Show more

The purpose of this study is to understand DMD functional losses or abilities and their association with independence and quality of life from the perspective of individuals with DMD and/or and their caregivers. This is a qualitative interview study in which individuals with DMD and/or their caregivers will be asked to participate in a semi-structured, approximately 60- minute interview. Interviews will focus on functional abilities and independence. Caregivers and boys with DMD will be interviewed. This study includes no treatment nor intervention; however, some participants are being treated by a drug that is approved in the U.S. and the U.K. and under investigation in other geographies.Show more

Italfarmaco is a private, family-owned pharmaceutical company founded in Milan in 1938 and still controlled by the De Santis family. It is larger than most people in the rare disease world realize: more than 3,800 employees, operations in over 90 countries, six manufacturing plants across Italy, Spain, Chile, and Brazil, and consolidated sales above EUR 1.1 billion in 2024, most of it from established medicines in women's health, cardiology, neurology, and hospital products. Francesco Di Marco, formerly of Boehringer Ingelheim and Amgen, became CEO in January 2026. In the United States the company operates through ITF Therapeutics, a rare disease affiliate set up in January 2024 in Concord, Massachusetts.

What put Italfarmaco on the map for patients is Duvyzat (givinostat), approved by the FDA on March 21, 2024 for Duchenne muscular dystrophy in patients 6 and older. Givinostat was discovered in Italfarmaco's own labs in collaboration with the Italian Telethon foundation and Duchenne Parent Project Italy, and it differs from most Duchenne drugs in one important way: it does not depend on which dystrophin mutation a boy has. It works by blocking enzymes called histone deacetylases (HDACs), which become overactive in Duchenne muscle and drive the inflammation, scarring, and fat replacement that follow the loss of dystrophin. In the Phase 3 EPIDYS trial, boys taking givinostat with steroids declined more slowly on a four-stair climb test over 18 months than boys on placebo, and MRI showed less muscle replaced by fat. Long-term follow-up presented in 2026 from 225 patients in the open-label extension found a median age at loss of ambulation of 17.3 years, compared with roughly 11 to 13 years in natural history studies. The main side effects to watch are low platelet counts, diarrhea, and raised triglycerides, so regular blood tests are part of treatment. Duvyzat is also approved in the United Kingdom (December 2024) and, conditionally for ambulant patients, in the European Union (June 2025); Health Canada granted priority review in June 2026, and JCR Pharmaceuticals licensed Japanese rights in December 2025.

The Duchenne program is still expanding. The Phase 3 ULYSSES trial (NCT05933057) is testing givinostat in boys and young men who can no longer walk, with results expected around 2028, and a Phase 2 study is establishing dosing for children ages 2 to 5. A U.S. registry called PROVIDUS is tracking 300 patients on the drug in real-world use. Outside Duchenne, givinostat is in a Phase 3 trial (GIV-IN PV, NCT06093672) against hydroxyurea in high-risk polycythemia vera, a rare blood cancer driven by the JAK2 mutation, where the drug holds FDA Fast Track designation. Italfarmaco also lists Becker muscular dystrophy and ALS among its rare disease programs, though a Phase 2 Becker study finished in 2021 and no new trial has been registered in either condition.

Italfarmaco has 1 FDA-approved drug with a rare disease use that we track, as of October 2026.

Duvyzat (givinostat), approved 2024: Duchenne Muscular Dystrophy.

Source: the FDA's openFDA label database, matched to the rare diseases on Trial Friend; the list updates automatically and can include older products.

Yes, for 1 of its FDA-approved rare disease medicine that we track, each checked on Italfarmaco's own website.

Duvyzat: ITF ARC, 1-855-448-3272. Offers copay help with private insurance, a free medicine program, a short-term supply while insurance is decided and insurance help. Checked September 24, 2026.

Medicare and Medicaid: drugmaker copay cards are limited to private insurance, so people with government coverage usually look to a free medicine program or a charity fund, both on the help paying page.

Eligibility is decided by each program, so call the number listed before a prescription is filled.