At Ipsen, we are breaking new ground in rare bone and liver diseases, delivering solutionsthat modify the course of rare diseases and improve the often-debilitating symptoms that impact dailylife.
Ipsen’s purpose in rare diseases
We are working to uncover the science behind rare diseases. We identify well-defined drugtargets to bring medicines that modify the course of rare diseases and alleviate symptomsto improve patients’ daily lives.
We are proud to have delivered the most extensive indications and medicines in rare liverdisease, and to have provided the first approved treatment for an ultra-rare bone disease.
Executing translational science and advancing targeted therapies, our research is focusedon the intersection between patient’s lived experiences and innovative science, to delivermeaningful 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.
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Advancing Science with Purpose
Ipsen is advancing medicines in oncology, rare diseases, and neurosciencethrough science-driven innovation. Explore our therapeutic areas to seehow we’re transforming innovation into medicine.