Ipsen’s purpose in rare diseases
We are working to uncover the science behind rare diseases. We identify well-defined drug targets to bring medicines that modify the course of rare diseases and alleviate symptoms to improve patients’ daily lives.
We are proud to have delivered extensive indications and medicines in rare liver disease, and to have provided the first approved treatment for an ultra-rare bone disease.
Executing translational science and advancing targeted therapies, our research is focused on the intersection between patient’s lived experiences and innovative science, to deliver meaningful medicines.
How Ipsen drives science with purpose in rare disease
Ipsen’s science in rare cholestatic liver disease
Using science to restore balance
Our focus is on expanding therapeutic possibilities for rare liver diseases where current options remain limited.
We are focused on five rare liver diseases: ALGS, BA, PBC, PFIC, and PSC, and aim to restore bile balance through modulating three different receptors involved in the bile acid system to relieve debilitating symptoms and improve long-term liver health.
IBAT and ASBT inhibitors reduce bile acid reabsorption in the intestine, lowering toxic bile buildup that contributes to pruritus and liver damage. PPAR agonists activate receptors in liver cells to regulate bile acid metabolism, reduce inflammation, and help protect against disease progression.
Our commitment extends beyond symptom relief – we strive to drive breakthroughs that reshape the future of treatment and redefine what’s possible for people with rare liver diseases.
Ipsen’s science in rare bone disease
Targeting the source of abnormal bone growth
We are advancing treatment innovation in fibrodysplasia ossificans progressiva (FOP), an ultra-rare and severely disabling genetic condition in which bone abnormally forms in soft tissues. This progressive disease leads to severe loss of mobility, breathing difficulties, and significantly shortened life expectancy.
FOP is caused by excessive activity in a receptor called ALK2, part of the bone morphogenetic protein (BMP) signaling pathway – a system that normally regulates healthy bone development.
Our first-of-its-kind research targets multiple points along the BMP pathway to help block the abnormal signals that drive the disease. By building on the first approved treatment and advancing new avenues of investigation, we aim to continue leading progress in FOP and make a lasting difference in the lives of patients and their families.
Meet the team
Discover the people driving R&D at Ipsen, advancing science with purpose all the way from discovery to patient impact.
Advancing Science with Purpose
We’re advancing medicines in oncology, rare diseases and neuroscience through science-led innovation. Explore our Neuroscience area to see how we’re transforming innovation into medicine.