Ipsen

Ipsen works on 17 rare diseases tracked on Trial Friend, including Acromegaly, Alagille Syndrome, Duchenne Muscular Dystrophy and 14 more, with 19 recruiting clinical trials and 5 FDA-approved rare disease drugs.

Ipsen is a French biopharmaceutical company focused on oncology, rare diseases, and neuroscience. Key rare disease products include Somatuline Depot (lanreotide) for acromegaly, Sohonos (palovarotene) for fibrodysplasia ossificans progressiva, Bylvay (odevixibat) for progressive familial intrahepatic cholestasis and Alagille syndrome pruritus, and Iqirvo (elafibranor) for primary biliary cholangitis (approved 2024). The company also has elafibranor in Phase 2 for primary sclerosing cholangitis.

Type
Diversified Pharma
Ticker
IPSEY
Headquarters
Paris, France
Founded
1929
Website
ipsen.com
19
Active Rare Disease Trials
5
Approved Rare Disease Drugs
17
Rare Diseases in Portfolio
97
Years Active

Focus areas at Ipsen

Within its broader pharmaceutical portfolio, Ipsen has active clinical trial programs and drug development efforts across 17 rare diseases, including Acromegaly, Alagille Syndrome, Duchenne Muscular Dystrophy, Fibrodysplasia Ossificans Progressiva, Friedreich Ataxia, and 12 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 Ipsen, 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.

Ipsen is headquartered in Paris, France, founded in 1929, publicly traded under the ticker symbol IPSEY. 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.

Ipsen Drug Pipeline

Ipsen has 19 active clinical trials across 5 development stages, with 19 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 Ipsen'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.

Understand Ipsen's pipeline
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1
Early Phase / Phase 11 trial
Relapsed or Refractory B-cell Non-Hodgkin Lymphoma
Recruiting
3
Phase 23 trials
Recruiting
Colorectal Adenocarcinoma+4 more
Recruiting
5
Phase 35 trials
1
Phase 4 / Post-Market1 trial
Tremor, Limb
Recruiting
9
Other9 trials
Spasticity as Sequela of Stroke
Recruiting
Advanced Renal Cell Carcinoma+1 more
Recruiting

Ipsen Clinical Trials (19)

Active and recruiting clinical trials sponsored by Ipsen, 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.

Ask about Ipsen's trials
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ACTIVE NOT RECRUITINGRecently updatedNCT06055725

A Study to Estimate How Often Post-stroke Spasticity Occurs and to Provide a Standard Guideline on the Best Way to Monitor Its Development

Spasticity as Sequela of Stroke

This study will monitor patients during the first year following their stroke. Stroke is a very serious condition where there is a sudden interruption of blood flow in the brain. The main aim of the study will be to find out how many of those who experience their first-ever stroke then go on to develop spasticity that would benefit from treatment with medication. Spasticity is a common post-stroke condition that causes stiff or ridged muscles. The results of this study will provide a standard guideline on the best way to monitor the development of post-stroke spasticity.

Ages 18 Years - 90 Years55 locations
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ACTIVE NOT RECRUITINGRecently updatedNCT07497724

Odevixibat Outcomes in Patients With PFIC Versus an External Control Cohort (OvEC-PFIC)

Progressive familial intrahepatic cholestasis (PFIC) is a rare inherited liver disease that causes a build-up of bile acids in the liver. This can lead to severe itching (pruritus), poor sleep, impaired growth, liver damage, and in some cases the need for surgery or liver transplantation. The purpose of this non-interventional, retrospective study is to compare long-term health outcomes in patients with PFIC. The comparison is between patients who received odevixibat in two odevixibat clinical trials (Studies A4250-005 and A4250-008) and an aligned, balanced external control cohort of patients with PFIC from the Natural course and Prognosis of PFIC and Effect of biliary Diversion (NAPPED) registry who were not treated with odevixibat (or other ileal bile acid transporter \[IBAT\] inhibitors). Outcomes such as liver transplantation, death, and surgical biliary diversion (SBD) will be examined to better understand how treatment with odevixibat compares to the natural course of PFIC. This study aims to provide a robust comparative evaluation of long-term clinical outcomes with odevixibat.

Ages 3 Months - 100 Years1 location
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ACTIVE NOT RECRUITINGPHASE3Recently updatedNCT04526665

Study of Elafibranor in Patients With Primary Biliary Cholangitis (PBC)

Intervention: Elafibranor 80mg, Placebo

The participants of this study will have confirmed Primary Biliary Cholangitis (PBC) with inadequate response or intolerance to ursodeoxycholic acid (which is a medication used in the management and treatment of cholestatic liver disease). PBC is a slowly progressive disease characterized by damage of the bile ducts in the liver, leading to a buildup of bile acids which causes further damage. The liver damage in PBC may lead to scarring (cirrhosis). PBC may also be associated with multiple symptoms. Many patients with PBC may require liver transplant or may die if the disease progresses and a liver transplant is not done. The main aim of this study is to determine if elafibranor (the study drug) is better than placebo (a dummy treatment) at decreasing the levels of a specific blood test (alkaline phosphatase) that provides information about participant's disease. This study will also evaluate the safety of long-term treatment with elafibranor, as well as the impact on symptoms such as itchy skin (pruritus) and tiredness (fatigue). This study has two main parts: Part 1 will compare a daily dose of elafibranor to a daily dose of placebo and will last between a minimum of one year and a maximum of two years. Part 2, all participants will receive elafibranor for a period of up to 5 years or until the total treatment duration (part 1 and part 2) reaches 6 years, whichever occurs first.

Ages 18 Years - 75 Years115 locations
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ACTIVE NOT RECRUITINGRecently updatedNCT03647878

Study of Cabozantinib as Monotherapy or in Combination With Nivolumab in Patients With Advanced or Metastatic Renal Cell Carcinoma Under Real-life Clinical Setting in 1st Line Treatment.

Advanced Renal Cell CarcinomaMetastatic Renal Cell Carcinoma

The purpose of the protocol, is to describe the use of cabozantinib tablets as monotherapy or in combination with nivolumab including the number of dose reductions, dose interruptions and terminations due to (serious) adverse events in subjects with advanced or metastatic renal cell carcinoma (mRCC) treated in real-life clinical setting in 1st line treatment.

Ages 18 Years+82 locations
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RECRUITINGPHASE3Recently updatedNCT06016842

A Long-Term Study of Elafibranor in Adult Participants With Primary Biliary Cholangitis

Intervention: Elafibranor, Matched 80 mg placebo

The participants of this study will have confirmed Primary Biliary Cholangitis (PBC) and cirrhosis (scarring of the liver). PBC is a slowly progressive disease, characterised by damage to the bile ducts in the liver, leading to a build-up of bile acids which causes further damage. The liver damage in PBC may lead to cirrhosis. PBC may also be associated with multiple symptoms. Many patients with PBC may require liver transplant or may die if the disease progresses and a liver transplant is not done. This study will compare a daily dose of elafibranor (the study drug) to a daily dose of placebo (a dummy treatment) and will last up to 3.5 years for each participant. The main aim of this study is to determine if elafibranor is better than placebo in preventing clinical outcome events showing disease worsening (including progression of disease leading to liver transplant or death). This study will also study the safety of long-term treatment with elafibranor, as well as the impact on symptoms such as itching and tiredness.

Ages 18 Years+186 locations
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ACTIVE NOT RECRUITINGPHASE3Recently updatedNCT05035030

Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome

Intervention: Odevixibat

The purpose of this study is to assess the long-term safety and effectiveness of odevixibat in participants with Alagille syndrome (ALGS). The participants of this study will have ALGS a rare genetic disorder that can affect multiple organ systems of the body including the liver, heart, skeleton, eyes and kidneys. Common symptoms, which often develop during the first three months of life, include blockage of the flow of bile from the liver (cholestasis), yellowing of the skin and mucous membranes (jaundice), poor weight gain and growth and severe itching (pruritis). The drug used for the study is odevixibat and was authorized for the treatment of cholestatic pruritus in infants with ALGS over 12 months of age by the United States Food and Drug Administration on 13 June 2023.

Ages not specified35 locations
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RECRUITINGRecently updatedNCT06850038

A Study Observing the Long-term, Effectiveness and Safety of Odevixibat (Bylvay) in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing Treatment

This study will collect information from patients with Alagille syndrome (ALGS) as they use odevixibat (Bylvay) in their daily lives. Odevixibat is a medicine that helps patients with ALGS, a rare disease that harms their liver and causes itching. The main aim of this study is to observe the long-term, everyday effectiveness and safety of the drug odevixibat in patients with ALGS who are receiving ongoing treatment.

Ages not specified10 locations
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Ipsen FDA-Approved Drugs (5)

Medications developed or marketed by Ipsen 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 NameBrand NameRare DiseasesApproval Date
ODEVIXIBAT
Ileal Bile Acid Transporter Inhibitor [EPC]
Bylvay
oral
Jul 20, 2021
MECASERMINIncrelex
subcutaneous
Aug 30, 2005
ELAFIBRANOR
Peroxisome Proliferator-activated Receptor Agonist [EPC]
IQIRVO
oral
Jun 10, 2024
IRINOTECAN HYDROCHLORIDEOnivyde
intravenous
Oct 22, 2015
LANREOTIDE ACETATESOMATULINE DEPOT
subcutaneous
Aug 30, 2007

Ipsen Trial Locations

Ipsen clinical trials are running at 685 sites in 37 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.

United States
205▼
Germany
108▼
Spain
43▼
Italy
42▼
France
36▼
United Kingdom
31▼
South Korea
28▼
Canada
20▼
Japan
16▼
Argentina
14▼
Poland
14▼
Chile
11▼

Rare Disease Focus Areas (17)

Diseases targeted by Ipsen's clinical trial and drug development programs

AcromegalyEndocrine & Hormonal

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...

Prevalence: Approximately 50-130 cases per million people; estimated 25,000-30,000 people in the United States with about 3,000 new cases diagnosed per year
Alagille SyndromeLiver & Hepatic

Alagille syndrome is a rare autosomal dominant disorder affecting the liver, heart, skeleton, face, and eyes. The condition results from mutations in genes regulating the Notch signaling pathway, lead...

Prevalence: 1 in 30,000 to 43,000 people
Duchenne Muscular DystrophyNeurological & Neuromuscular

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...

Prevalence: 1 in 3,500 to 5,000 male births
Fibrodysplasia Ossificans ProgressivaConnective Tissue & Musculoskeletal

Fibrodysplasia Ossificans Progressiva (FOP) is an extremely rare genetic disorder characterized by progressive heterotopic ossification, where soft tissues (muscles, tendons, ligaments) gradually tran...

Prevalence: Extremely rare, approximately 1 per 2 million people globally; extremely small patient population
Friedreich AtaxiaNeurological & Neuromuscular

Friedreich ataxia is an autosomal recessive neurodegenerative disease causing progressive damage to the nervous system, resulting in loss of coordination (ataxia), weakness, and heart problems. The co...

Prevalence: 1 in 50,000 people
Hemophilia ABlood & Immune

Hemophilia A is an X-linked bleeding disorder caused by deficiency or dysfunction of clotting factor VIII. Severity depends on factor levels, ranging from mild to severe hemorrhage. Modern factor repl...

Prevalence: 1 in 4,000 to 5,000 males worldwide; very rare in females

Patient Resources

Organizations and resources related to Ipsen's rare disease focus areas

Frequently Asked Questions About Ipsen

Common questions about Ipsen's rare disease programs, clinical trials, and treatments.