Recordati Rare Diseases
Recordati Rare Diseases works on 7 rare diseases tracked on Trial Friend, including Acromegaly, Cold Agglutinin Disease, Cushing Disease and 4 more, with 5 recruiting clinical trials and 9 FDA-approved rare disease drugs.
Recordati is an Italian pharmaceutical company with a dedicated rare disease division. Key products include Enjaymo (sutimlimab) for cold agglutinin disease (acquired from Sanofi in 2024 for $825 million), Signifor LAR (pasireotide) for acromegaly, Carbaglu (carglumic acid) for urea cycle disorders, and Cystadane (betaine) for homocystinuria.
Focus areas at Recordati Rare Diseases
Within its broader pharmaceutical portfolio, Recordati Rare Diseases has active clinical trial programs and drug development efforts across 7 rare diseases, including Acromegaly, Cold Agglutinin Disease, Cushing Disease, Homocystinuria, Methylmalonic Acidemia, and 2 additional rare conditions. These programs may span orphan drug designation, novel therapeutic mechanisms, and precision medicine approaches targeting the underlying causes of each disease.
The clinical trials section below shows all active and recruiting studies sponsored by Recordati Rare Diseases, sourced live from ClinicalTrials.gov. Each trial includes its current recruitment status, study phase (Phase 1 through Phase 4), conditions under investigation, and the number of active trial sites. The FDA-approved drugs section lists treatments that have received U.S. Food and Drug Administration approval, with brand names, generic names, approval dates, and matched rare disease indications from the openFDA database.
Recordati Rare Diseases is headquartered in Milan, Italy, founded in 1926, publicly traded under the ticker symbol REC. The company maintains a dedicated rare disease division alongside its broader therapeutic portfolio, working to bring innovative treatments to patients with conditions that have historically had limited or no treatment options.
Recordati Rare Diseases Drug Pipeline
Recordati Rare Diseases has 5 active clinical trials across 3 development stages, with 5 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 Recordati Rare Diseases'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.
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.
Recordati Rare Diseases FDA-Approved Drugs (9)
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 | |
| SUTIMLIMAB-JOME Classical Complement Pathway Inhibitor [EPC] | Enjaymo intravenous | Feb 4, 2022 | |
| OSILODROSTAT | Isturisa oral | — | Mar 6, 2020 |
| IBUPROFEN LYSINE | NEOPROFEN intravenous | Apr 13, 2006 |
Recordati Rare Diseases Trial Locations
Recordati Rare Diseases clinical trials are running at 107 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 (7)
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 autoantibodies that bind red blood cells in cold temperatures. The condition leads to intravascular hemolysis particularly i...
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...
Paragangliomas are rare neuroendocrine tumors arising from chromaffin tissue outside the adrenal medulla. Often hereditary with SDH mutations, they can produce catecholamines or other hormones. Treatm...
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.