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Recordati Rare Diseases

Drugmaker with rare disease work · based in Milan, Italy · founded 1926 · ticker REC · recordati.com

Recordati Rare Diseases works on 12 rare diseases we track, including Acromegaly, Acute Hepatic Porphyria and 10 more. It has 5 active clinical trials, and 4 are looking for volunteers now. It also has 8 FDA-approved rare disease drugs.

Recordati is an Italian pharmaceutical company with a dedicated rare disease division.

Recordati Rare Diseases pipeline

Each row lists the diseases Recordati Rare Diseases is studying at that stage, from the ones closest to approval down to the earliest work.

Approved
Drugs the FDA has approved.
Phase 2
Mid-size studies that check whether a drug works and at what dose.

Multiple Myeloma recruiting

Phase 1
First studies in people, mostly checking safety.

Non-Hodgkin Lymphoma recruiting

Other studies
Studies with no phase listed, such as registries and natural history studies.

Recordati Rare Diseases clinical trials

These are Recordati Rare Diseases trials for the rare diseases we track, from ClinicalTrials.gov. Statuses there can lag behind company news, so confirm with the study team before you plan around one.

4 trials looking for volunteers now

Looking for volunteersPhase 2Recently updatedNCT06679829

A Study of Melphalan With or Without Siltuximab in People With Multiple Myeloma Having an Autologous Stem Cell Transplant

Tests Siltuximab, Melphalan

For Multiple Myeloma

The purpose of this study is to see if siltuximab plus population pharmacokinetic (PK)-dosed melphalan works as well as the usual approach (body surface area \[BSA\]-dosed melphalan) in people with multiple myeloma (MM) who are receiving an autologous stem cell transplant (ASCT) as part of their standard treatment. The researchers will also see if siltuximab in combination with population PK-dosed melphalan works to decrease symptoms after an ASCT, and will study the safety of siltuximab. For the run-in, 15 patients will receive siltuximab, 11 mg/kg, seven days before and 14 days after autologous hematopoietic stem cell infusion (+/-2 day).

Ages 60 Years+7 locations
View full study
Looking for volunteersPhase 1Updated a few months agoNCT06447376

Study of Cytokine Release Syndrome Prophylaxis and Treatment With Siltuximab Prior to Epcoritamab

Tests Siltuximab, Epcoritamab, Gemcitabine and oxaliplatin

For Non-Hodgkin Lymphoma and Cytokine Release Syndrome

The goal of this clinical trial is to is to determine the safety, feasibility and efficacy of siltuximab prophylaxis of cytokine release syndrome and neurotoxicity occurring after epcoritamab subcutaneous administration for participants with large b-cell lymphoma (DLBCL) or follicular lymphoma (FL). Participants will receive siltuximab, prior to the injection of epcoritamab. Epcoritamab is administered in 28 day cycles for one year. After this injection, the physician will continue to watch participants for side effects and follow the condition for a minimum of 60 days.

Ages 18 Years - 65 Years1 location
View full study
Looking for volunteersHasn't posted an update in over a yearNCT05040178

An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics

Tests Carglumic Acid

For Hyperammonemia, Methylmalonic Acidemia and Propionic Acidemia

To obtain short-term and long-term clinical safety information, in pediatric and adult patients with PA and MMA treated with Carbaglu®.

Ages not specified5 locations
View full study
Looking for volunteersHasn't posted an update in over a yearNCT04176523

Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach

Tests Carglumic Acid

For Methylmalonic Acidemia and Propionic Acidemia

This is a prospective mixed-design study focused on the long-term management of propionic aciduria (PA) and methylmalonic aciduria (MMA) with N-carbamylglutamate (NCG) maintenance therapy. Treatment characteristics, clinical outcomes, and healthcare utilization data of patients diagnosed PA or MMA treated \>6 months therapy with NCG are collected at baseline, 12 months, 18 months, 36 months and 54 months. Qualitative interviews with adult patients and caregivers are conducted \>6 months after study enrollment to gain a better understanding of the disease burden and the treatment burden of patients and their families.

Ages 6 Months - 99 Years32 locations
View full study

1 trial underway, not taking new volunteers

Underway, not taking new volunteersNo updates in a whileNCT05791708

Cold Agglutinin Disease Real World Evidence Registry

Tests Sutimlimab

For Cold Agglutinin Disease (CAD) and Cold Agglutinin Syndrome (CAS)

This is a multinational, multi-center, observational, prospective, longitudinal disease registry designed to collect data on participants with cold agglutinin disease (CAD) or cold agglutinin syndrome (CAS). Among them, a minimum of 30 patients with CAD treated with sutimlimab are expected to take part in the sutimlimab cohort study. Patients with CAD who have been enrolled in previous sutimlimab clinical trials (e.g., BIVV009-01/LTS16214 \[NCT02502903,CAD patients\], BIVV009-03/EFC16215 \[NCT03347396\], and BIVV009-04/EFC16216 \[NCT03347422\]) and who either completed or discontinued the corresponding clinical trial are eligible to participate in the registry.

Ages 18 Years+68 locations
View full study
Ask about Recordati Rare Diseases's trials
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Follow a condition Recordati Rare Diseases is studying

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You'll get, for Acromegaly:
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  • Disease Updates, FDA decisions and condition news, usually 1 or 2 emails a month
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FDA-approved drugs from Recordati Rare Diseases

From the openFDA database. Tap a drug for side effects, dosing and help paying.

Carbaglu carglumic acid

For Urea Cycle Disorders and Methylmalonic Acidemia. Approved Mar 18, 2010. Taken by mouth.

Cystadane betaine

For Homocystinuria. Approved Oct 25, 1996. Taken by mouth.

Cystadrops cysteamine hydrochloride

For Cystinosis. Approved Aug 19, 2020. Taken by ophthalmic route.

Enjaymo sutimlimab-jome

For Cold Agglutinin Disease. Approved Feb 4, 2022. Taken by intravenous route.

Isturisa osilodrostat

Approved Mar 6, 2020. Taken by mouth.

Signifor pasireotide

For Cushing Disease. Approved Dec 14, 2012. Taken by subcutaneous route.

Signifor LAR pasireotide

For Acromegaly and Cushing Disease. Approved Dec 15, 2014.

Panhematin hemin

For Acute Hepatic Porphyria. Taken by intravenous route.

More about Recordati Rare Diseases

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 recurrent attacks of acute intermittent porphyria tied to the menstrual cycle; Carbaglu (carglumic acid) for high ammonia from NAGS deficiency, propionic acidemia or methylmalonic acidemia; and Cystadane (betaine) for homocystinuria.

Where Recordati Rare Diseases trials take place

The trials above run at 113 study sites in 10 countries. Tap a country to see its states, cities and hospitals.

United States
32▼
France
24▼
United Kingdom
18▼
Italy
16▼
Spain
13▼
Germany
4▼
Austria
2▼
Japan
2▼
Norway
1▼
Sweden
1▼

Rare diseases Recordati Rare Diseases works on

Each disease page lists every open trial, not only Recordati Rare Diseases's, plus approved treatments.

Acromegaly

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.

How common: About 50 to 70 people per million, according to NORD, with 3 to 11 new cases per million people each year

Acute Hepatic Porphyria

Acute hepatic porphyria (AHP) is a group of 4 rare inherited disorders of heme production in the liver: acute intermittent porphyria, hereditary coproporphyria, variegate porphyria and ALAD...

How common: Symptomatic acute intermittent porphyria, the most common type, affects about 5 to 6 people per million in Europe (NORD cites 5.9 per million; a European prospective study found 5.4 per million), roughly 1 in 170,000 to 1 in 185,000 people, and the rate is thought to be similar in the United States

Cold Agglutinin Disease

Cold agglutinin disease is a rare autoimmune hemolytic anemia caused by IgM antibodies that stick to red blood cells in the cold.

How common: About 16 per million people for primary CAD in a Norwegian population study, roughly 1 in 60,000, with about 1 new case per million people each year

Cushing Disease

Cushing Disease is a rare endocrine disorder caused by an ACTH-secreting pituitary adenoma that leads to excessive cortisol production.

How common: Estimated 2-8 new cases per million people per year, though recent population studies suggest the true incidence may be higher

Cystinosis

Cystinosis is an inherited condition in which the amino acid cystine builds up inside cells and forms crystals that damage organs, especially the kidneys and eyes.

How common: About 1 in 100,000 to 200,000 newborns worldwide (MedlinePlus Genetics), and about 1 in 26,000 in Brittany, France

Gastroparesis

Gastroparesis is a condition in which the stomach empties food too slowly even though nothing is blocking it, causing early fullness, nausea, vomiting, bloating and upper belly pain.

How common: In a Minnesota population study cited by the NIDDK, about 10 men and 40 women per 100,000 had diagnosed gastroparesis

Homocystinuria

Homocystinuria is a rare inherited metabolic disorder characterized by elevated homocysteine levels in blood and urine.

How common: About 1 in 344,000 people worldwide for CBS deficiency, according to NORD, and higher in some countries, such as about 1 in 65,000 in Ireland

Methylmalonic Acidemia

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.

How common: 1 in 50,000 to 100,000 live births in the U.S.; identified through newborn screening in all 50 states

Multiple Myeloma

Multiple myeloma is a cancer of plasma cells, the white blood cells that make antibodies.

How common: About 36,000 new US diagnoses expected in 2026 and roughly 203,000 people living with it, which puts myeloma right at the edge of the US rare disease threshold; it is 1.7% of all new cancers (NCI SEER)

Neuroendocrine Tumors

Neuroendocrine tumors (NETs) grow from hormone-making cells, most often in the small intestine, pancreas, rectum or lungs.

How common: About 244,000 people in the US were living with a neuroendocrine neoplasm, the wider group that includes NETs, in 2021, with about 8.5 new diagnoses per 100,000 people a year (Dasari et al., JAMA Network Open 2025); an earlier estimate for NETs alone was about 171,000 in 2014 (JAMA Oncology 2017)

Paraganglioma

Paragangliomas are rare neuroendocrine tumors arising from chromaffin tissue outside the adrenal medulla.

How common: 1 in 300,000 to 1,000,000; ~30% are hereditary

Urea Cycle Disorders

Urea Cycle Disorders (UCDs) are a group of genetic conditions affecting the enzymes that convert toxic ammonia to urea for excretion.

How common: About 1 in 35,000 births in the United States, based on newborn screening and registry data (Summar et al., 2013); Ornithine Transcarbamylase (OTC) deficiency is most common form

Where to get support

Patient groups can explain a diagnosis, point you to specialists and tell you about trials before they open. Recordati Rare Diseases's own site is recordati.com, and our help paying for treatment page lists drugmaker programs and charity funds.

Questions about Recordati Rare Diseases