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About Sucampo Glossary

About Sucampo:

Sucampo Pharmaceuticals, Inc. is focused on the development and commercialization of specialized medicines that meet major unmet medical needs of patients worldwide. Sucampo has two marketed products – AMITIZA, its lead product, and RESCULA – and a late-stage pipeline of product candidates in clinical development for orphan disease areas. VTS-270 is a mixture of 2-hydroxypropyl-B-cyclodextrins with a specific compositional fingerprint that has been granted orphan designation in the U.S. and Europe and is in a pivotal Phase 2/3 clinical trial for the treatment of Niemann-Pick Disease Type C-1. Sucampo has an option for the North American rights to CPP1- x/sulindac, which is in Phase 3 development for the treatment of familial adenomatous polyposis and has been granted orphan drug designation in the U.S. A global company, Sucampo is headquartered in Rockville, Maryland, and has operations in Japan and Switzerland. For more information, please visit

Glossary of Terms:

ASMD  –  Acid Sphingomyelinase (Niemann-Pick Type A/B & Type B)
EMA  –  European Medicines Agency (European equivalent of FDA)
ERT  –  
Enzyme Replacement Therapy
FDA  –  
United States Food and Drug Administration

HDAC  –  Histone Deacetylase Inhibitors
ICV  –  Intracerebroventricular
IND  –  Investigational New Drug
i-IND  –  Individual Investiational New Drug
IRB  –  Institutional Review Board
NICHD  –  National Institute of Child Health and Human Development
NIH  –  National Institutes of Health
NPC  –  Niemann-Pick Disease Type C Disease
TRND  –  Therapeutics for Rare and Neglected Diseases


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