Sobi's Orfadin oral suspension for HT-1 approved in USA

Swedish Orphan Biovitrum (STO: SOBI) has gained approval from the US Food and Drug Administration for Orfadin (nitisinone) oral suspension to treat hereditary tyrosinaemia type-1 (HT-1), in combination with dietary restriction of tyrosine and phenylanine.

HT-1 is a rare genetic disease that affects infants and children. It can result in liver and kidney complications, and is potentially fatal if untreated. Some 20 years ago, before pharmacological treatment was available, fewer than one third of infants diagnosed with HT-1 before two months of age, lived past their second birthday.

Today, treatment with Orfadin as an adjunct to dietary restriction as well as early diagnosis have improved outcomes for HT-1 patients.