Italfarmaco
Italfarmaco works on 5 rare diseases tracked on Trial Friend, including Acromegaly, Becker Muscular Dystrophy, Duchenne Muscular Dystrophy and 2 more, with 6 recruiting clinical trials and 1 FDA-approved rare disease drug.
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 Drug Pipeline
Italfarmaco has 6 active clinical trials across 3 development stages, with 6 currently recruiting participants. Clinical trials advance through phases: Phase 1 tests safety in a small group, Phase 2 evaluates effectiveness and side effects, Phase 3 confirms benefit in a larger population, and Phase 4 monitors long-term safety after FDA approval.
Note: This pipeline counts the active and recruiting trials listed on this page, from ClinicalTrials.gov. For companies that work outside rare disease, we list only trials for rare diseases we track, so the company's full pipeline may be larger.
Italfarmaco Clinical Trials (6)
Active and recruiting clinical trials sponsored by Italfarmaco, sourced live from ClinicalTrials.gov. Each trial card shows the study phase, current recruitment status, conditions under investigation, study locations, eligibility criteria, and a direct link to the full ClinicalTrials.gov record. You can also download a one-page PDF summary to share with your doctor.
Note: Recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator.
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Italfarmaco FDA-Approved Drugs (1)
Medications developed or marketed by Italfarmaco that have received U.S. Food and Drug Administration approval. Drug data is sourced from the openFDA database and includes brand names, generic names, approval dates, and matched rare disease indications. Where a drug treats a condition covered by Trial Friend, the disease name links directly to that disease page.
| Drug Name | Brand Name | Rare Diseases | Approval Date |
|---|---|---|---|
| GIVINOSTAT Histone Deacetylase Inhibitor [EPC] | DUVYZAT oral | Mar 21, 2024 |
Italfarmaco Trial Locations
Italfarmaco clinical trials are running at 177 sites in 20 countries. Click any country to drill down by state, city, and individual research facility. Proximity to a trial site is one of the most important factors in deciding whether to participate.
Rare Disease Focus Areas (5)
Diseases targeted by Italfarmaco's clinical trial and drug development programs
Acromegaly is a rare hormonal disorder caused by excessive growth hormone production, typically from a pituitary adenoma, resulting in abnormal growth of hands, feet, and facial features. It also caus...
Becker Muscular Dystrophy (BMD) is an X-linked inherited muscular dystrophy caused by mutations in the dystrophin gene that produce a partially functional dystrophin protein. BMD is milder than Duchen...
Duchenne muscular dystrophy is an X-linked genetic disorder causing progressive muscle weakness and degeneration, beginning in early childhood. The defective dystrophin protein normally protects muscl...
Hodgkin lymphoma is a cancer of the lymphatic system characterized by the presence of abnormal Reed-Sternberg cells. It typically starts in lymph nodes in the neck, chest, or armpits and can spread to...
Polycythemia Vera is a myeloproliferative neoplasm in which bone marrow produces too many red blood cells, leading to thick blood that carries oxygen less effectively and increases clot risk. Most pat...
Patient Resources
Organizations and resources related to Italfarmaco's rare disease focus areas
Frequently Asked Questions About Italfarmaco
Common questions about Italfarmaco's rare disease programs, clinical trials, and treatments.