Early treatment and a longer life - new Orfadin formats meet new patient needs

Swedish Orphan Biovitrum AB (publ) (Sobi) announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion for the oral suspension formulation and the 20mg capsule of Orfadin (nitisinone) for the treatment of hereditary tyrosinaemia type-1 (HT-1). HT-1 is a rare genetic disease that affects infants and children; it is progressive in nature, may result in liver and kidney failure and can be fatal if untreated.
Source: Pharmacy Europe - Category: Drugs & Pharmacology Authors: Tags: *** Editor's Pick Practical therapeutics Industry News Legislation & regulation Hepatic disorders Nephrology chmp EMA genetic disease hereditary tyrosinaemia type-1 HT-1 Latest News nitisinone oral suspension orfadin paed Source Type: news