ExCEEd Orphan Distribution d.o.o., a Croatia based fully-owned subsidiary of ExCEEd Orphan s.r.o., a company providing full business solutions for the treatment of rare diseases, signed an exclusive agreement with Mirum Pharmaceuticals, Inc., a biopharmaceutical company dedicated to developing treatments for rare liver diseases for the distribution and marketing rights to LIVMARLI™ (maralixibat) oral solution in six countries throughout Central and Eastern European (CEE), including Croatia, Czechia, Hungary, Poland, Slovakia, and Slovenia.
The FDA recently granted marketing approval in the United States for LIVMARLI as the first and the only approved therapy for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) one year of age and older. The European marketing authorisation application for LIVMARLI for the treatment of cholestatic liver disease in patients with ALGS is already in the process and is expected to be completed in the second half of 2022.
Jiri Hermanek, Chief Executive Officer and Founding Partner of ExCEEd Orphan, commented:
“We are very pleased to announce this collaboration to make LIVMARLI available to patients suffering from ALGS in our countries. Should LIVMARLI receive approval by the EMA, patients will have a new innovative treatment to address their disease, which, outside of the U.S., there are no available treatments and many patients succumb to a liver transplantation. LIVMARLI® will represent an important addition to continuously growing portfolio of rare disease medications marketed and distributed by ExCEEd Orphan and we are fully committed to bringing this unique medication to all patients in needs in all respective countries.”
About LIVMARLI™ (maralixibat) oral solution
LIVMARLI™ (maralixibat) oral solution is an orally administered, once-daily, ileal bile acid transporter (IBAT) inhibitor approved by the U.S. Food and Drug Administration for the treatment of cholestatic pruritus in patients with Alagille syndrome one year of age and older.
About Alagille syndrome
Alagille syndrome (ALGS) is a rare genetic disorder in which bile ducts are abnormally narrow, malformed and reduced in number, which leads to bile accumulation in the liver and ultimately progressive liver disease. The estimated incidence of ALGS is one in every 30,000 people. In patients with ALGS, multiple organ systems may be affected by the mutation, including the liver, heart, kidneys and central nervous system. The accumulation of bile acids prevents the liver from working properly to eliminate waste from the bloodstream and, according to recent reports, 60% to 75% of patients with ALGS have a liver transplant before reaching adulthood. Signs and symptoms arising from liver damage in ALGS may include jaundice (yellowing of the skin), xanthomas (disfiguring cholesterol deposits under the skin), and pruritus (itch)2. The pruritus experienced by patients with ALGS is among the most severe in any chronic liver disease and is present in most affected children by the third year of life.
About ExCEEd Orphan s.r.o.
ExCEEd Orphan was founded in 2018 by five rare disease experts from multiple Central and Eastern European (CEE) countries. The Company is focusing on innovative treatments for rare diseases and has extensive experience in launching innovative medicines in this field. The portfolio of ExCEEd Orphan includes products in therapeutic areas like haematology, neurology, immunology, and metabolic diseases.
For further information about ExCEEd Orphan, please visit
About Mirum Pharmaceuticals, Inc.
Mirum’s late-stage pipeline includes maralixibat (LIVMARLI), an oral ileal bile acid transporter (IBAT) inhibitor being studied in progressive familial intrahepatic cholestasis and biliary atresia, and volixibat, also an oral IBAT inhibitor, being evaluated in intrahepatic cholestasis of pregnancy and primary sclerosing cholangitis. Mirum has also acquired the exclusive option to develop and commercialize gene therapy programs VTX-803 and VTX-802 for PFIC3 and PFIC2, respectively, from Vivet Therapeutics SAS, following preclinical evaluation and investigational new drug-enabling studies.