European Medicines Agency grants lead drug A4250 for orphan medicine for daredevil atresia
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Last Update: 2020-06-10
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Source: Internet
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Author: User
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bile duct atresia is a rare and life-threatening liver disease, and there is no approved treatmentdrug(http://recently, theof the http:// thecompany of Albireo Pharma, announcedthat the EuropeanMedicines(http://Authority (EMA) had granted its lead drug, the A4250, the right to treat orphans with dystoinAbout A4250
A4250 is the first highly effective selective reclining bile acid transporter (IBAT) inhibitor with minimal systemic exposure and local functioning in the intestine, A4250 is currently being developed for the treatment of rare children's bile siltliver liver disease, the first targeted indication is PFIC, which is currently in Phase III clinical treatment pFIC (type 1 and type 2)in the United States and the European Union, A4250 has previously been granted the right to treat orphans for progenitive family-based intra-hepatic bile siltation (PFIC), Alagille syndrome and primary bile ductitis (PBC)in the United States, the A4250 has also been granted a rare paediatric disease and fast-track status;
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