Recordati Rare Diseases
Recordati Rare Diseases works on 8 rare diseases tracked on Trial Friend, including Acromegaly, Cold Agglutinin Disease, Cushing Disease and 5 more, with 5 active clinical trials (4 recruiting) and 8 FDA-approved rare disease drugs.
Recordati is an Italian pharmaceutical company with a dedicated rare disease division. Its US rare disease medicines include Enjaymo (sutimlimab) for cold agglutinin disease, bought from Sanofi in 2024 for $825 million upfront; Isturisa (osilodrostat) for Cushing's syndrome; Signifor LAR (pasireotide) for acromegaly and Cushing's disease; Sylvant (siltuximab) for multicentric Castleman disease; Panhematin (hemin) for attacks of acute intermittent porphyria; Carbaglu (carglumic acid) for high ammonia from NAGS deficiency, propionic acidemia or methylmalonic acidemia; and Cystadane (betaine) for homocystinuria.
Recordati Rare Diseases Drug Pipeline
Recordati Rare Diseases has 5 active clinical trials across 3 development stages, with 4 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.
Recordati Rare Diseases Clinical Trials (5)
Active and recruiting clinical trials sponsored by Recordati Rare Diseases, 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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Recordati Rare Diseases FDA-Approved Drugs (8)
Medications developed or marketed by Recordati Rare Diseases 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 |
|---|---|---|---|
| CARGLUMIC ACID Carbamoyl Phosphate Synthetase 1 Activator [EPC] | Carbaglu oral | Mar 18, 2010 | |
| BETAINE Methylating Agent [EPC] | Cystadane oral | Oct 25, 1996 | |
| CYSTEAMINE HYDROCHLORIDE | CYSTADROPS ophthalmic | Aug 19, 2020 | |
| SUTIMLIMAB-JOME Classical Complement Pathway Inhibitor [EPC] | Enjaymo intravenous | Feb 4, 2022 | |
| OSILODROSTAT | Isturisa oral | — | Mar 6, 2020 |
Recordati Rare Diseases Trial Locations
Recordati Rare Diseases clinical trials are running at 113 sites in 10 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 (8)
Diseases targeted by Recordati Rare Diseases'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...
Cold agglutinin disease is a rare autoimmune hemolytic anemia caused by IgM antibodies that stick to red blood cells in the cold. Most of the red cell destruction happens outside the blood vessels, ma...
Cushing Disease is a rare endocrine disorder caused by an ACTH-secreting pituitary adenoma that leads to excessive cortisol production. It causes distinctive changes including central obesity with pur...
Homocystinuria is a rare inherited metabolic disorder characterized by elevated homocysteine levels in blood and urine. 3 biochemically distinct types exist; the most common involves cystathionine bet...
Methylmalonic acidemia is a group of inherited metabolic disorders in which the body cannot properly break down certain proteins and fats, leading to toxic buildup of methylmalonic acid. The most comm...
Multiple myeloma is a cancer of plasma cells, the white blood cells that make antibodies. Abnormal plasma cells crowd the bone marrow, weaken bones and release a useless protein that can damage the ki...
Patient Resources
Organizations and resources related to Recordati Rare Diseases's rare disease focus areas
Frequently Asked Questions About Recordati Rare Diseases
Common questions about Recordati Rare Diseases's rare disease programs, clinical trials, and treatments.