Chiesi Farmaceutici
Chiesi Farmaceutici works on 13 rare diseases tracked on Trial Friend, including Acromegaly, Alpha-1 Antitrypsin Deficiency, Cystic Fibrosis and 10 more, with 9 recruiting clinical trials and 9 FDA-approved rare disease drugs.
Chiesi is an international biopharmaceutical company with a broad rare disease portfolio. Key products include Lamzede (velmanase alfa) for alfa-mannosidosis, Elfabrio (pegunigalsidase alfa) for Fabry disease, FILSUVEZ for epidermolysis bullosa, JUXTAPID/LOJUXTA (lomitapide) for homozygous familial hypercholesterolemia, Mycapssa (octreotide) for acromegaly, and FERRIPROX (deferiprone) for transfusional iron overload in sickle cell disease and thalassemia. The company also has programs in cystic fibrosis and pulmonary fibrosis.
Chiesi Farmaceutici Drug Pipeline
Chiesi Farmaceutici has 9 active clinical trials across 3 development stages, with 9 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 includes all of Chiesi Farmaceutici's active interventional trials, not only those targeting rare diseases. We show the full pipeline because a company's broader research activity, therapeutic expertise, and development infrastructure directly shape its ability to advance rare disease programs. A strong overall pipeline often signals deeper clinical operations, faster enrollment capabilities, and greater commitment to bringing new treatments to patients.
Chiesi Farmaceutici Clinical Trials (9)
Active and recruiting clinical trials sponsored by Chiesi Farmaceutici, 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.
Chiesi Farmaceutici FDA-Approved Drugs (9)
Medications developed or marketed by Chiesi Farmaceutici 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 |
|---|---|---|---|
| TOBRAMYCIN Aminoglycoside Antibacterial [EPC] | BETHKIS respiratory (inhalation) | Oct 12, 2012 | |
| PEGUNIGALSIDASE ALFA Hydrolytic Lysosomal Neutral Glycosphingolipid-specific Enzyme [EPC] | ELFABRIO intravenous | May 9, 2023 | |
| DEFERIPRONE Iron Chelator [EPC] | FERRIPROX oral | Oct 14, 2011 | |
| BIRCH TRITERPENES | FILSUVEZ topical | Dec 18, 2023 | |
| LOMITAPIDE MESYLATE | Juxtapid oral | Dec 21, 2012 |
Chiesi Farmaceutici Trial Locations
Chiesi Farmaceutici clinical trials are running at 66 sites in 12 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 (13)
Diseases targeted by Chiesi Farmaceutici'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...
Alpha-1 antitrypsin deficiency is a genetic disorder affecting the lungs and liver, caused by insufficient production of the protective enzyme alpha-1 antitrypsin. Without adequate protection, neutrop...
Cystic fibrosis is an autosomal recessive genetic disorder affecting the CFTR protein, which normally regulates chloride transport. Defective CFTR causes thick, sticky secretions in the lungs and dige...
Epidermolysis bullosa is a group of rare genetic blistering disorders caused by mutations affecting proteins anchoring the epidermis to the dermis. Fragile skin blisters and erodes with minimal trauma...
Fabry disease is a rare inherited lysosomal storage disorder where a missing enzyme causes fatty substances called globotriaosylceramide to accumulate in cells throughout the body. This buildup damage...
Familial Chylomicronemia Syndrome is a rare inherited metabolic disorder where the body cannot break down certain fats (triglycerides) normally, causing dangerously high blood fat levels. People with ...
Patient Resources
Organizations and resources related to Chiesi Farmaceutici's rare disease focus areas
Frequently Asked Questions About Chiesi Farmaceutici
Common questions about Chiesi Farmaceutici's rare disease programs, clinical trials, and treatments.