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PFIC is a group of inherited liver diseases resulting from mutations in genes that impact bile secretion that are progressive and life-threatening. Typical onset of PFIC is in infancy or early childhood with a hallmark feature of this condition being severe and debilitating pruritus (itching), which has been shown to considerably reduce quality of life and can result in the need for liver transplantation.[i], [ii]The estimated global incidence of PFIC is one in 50,000 to 100,000 people worldwide,[iii] resulting in a designation of this condition as extremely or ultra rare.[iv]
About Medison Pharma
Medison is a global pharma company focused on providing access to highly innovative therapies to patients in international markets. Medison is the first to create an international commercialization platform for highly innovative therapies, helping to save and improve lives by making the best available novel treatments accessible to patients in international markets. Medison has a track record of multi-territorial partnerships with leading pharmaceutical and biotech companies seeking to expand their global reach. To learn more visit www.medisonpharma.com
About Ipsen
Ipsen is a global, mid-sized biopharmaceutical company focused on transformative medicines in Oncology, Rare Disease and Neuroscience. With total sales of €3.0bn in FY 2022, Ipsen sells medicines in over 100 countries. Alongside its external-innovation strategy, the Company’s research and development efforts are focused on its innovative and differentiated technological platforms located in the heart of leading biotechnological and life-science hubs: Paris-Saclay, France; Oxford, U.K.; Cambridge, U.S.; Shanghai, China. Ipsen has around 5,400 colleagues worldwide and is listed in Paris (Euronext: IPN) and in the U.S. through a Sponsored Level I American Depositary Receipt program (ADR: IPSEY). For more information, visit ipsen.com
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Ipsen’s forward-looking statements
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[i] Progressive Familial Intrahepatic Cholestasis Advocacy and Resource Network. Learn About PFIC Disease.https://www.pfic.org/learn-about-pfic-disease/. Accessed October 5, 2023.
[ii] Thompson, R., Arnell, H., et. al. Odevixibat treatment in progressive familial intrahepatic cholestasis: a randomized, placebo-controlled, phase 3 study. Lancet Gastroenterol Hepatol 2022; 7: 830–42.
[iii] Progressive Familial Intrahepatic Cholestasis Advocacy and Resource Network. Learn About PFIC Disease.https://www.pfic.org/learn-about-pfic-disease/. Accessed October 5, 2023.
[iv] Smith, C.I., Bergman, P., Hagey, D. Estimating the number of diseases – the concept of rare, ultra-rare, and hyper-rare. iScience. 2022; Aug 19; 25(8): 104698.
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