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Sobi’s Orfadin approved in the US


Swedish Orphan Biovitrum announces that the Food and Drug Administration (FDA) has approved a higher strength 20 mg 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 and may result in liver and kidney complications and can be fatal if untreated.

Twenty 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.[i] Today, treatment with Orfadin as an adjunct to dietary restriction of tyrosine and phenylalanine as well as early diagnosis have improved outcomes for HT-1 patients. Today there are people with HT-1 diagnosed early and started on treatment early in life, growing up to become adults.

Because dosing is adjusted by weight, patients need progressively higher doses as they grow. Sobi has developed the higher capsule strength of 20 mg to support the treatment regimens of adolescent and adult patients that may allow for fewer capsules per dose. This is important because of the chronic nature of their treatment.

“The HT-1 patient journey has driven Sobi’s development of a new strength for Orfadin in order to support sustainable outcomes over a lifetime for HT-1 patients. We are committed to the HT-1 community and are proud to expand the treatment alternatives for this community,” says Michael Yeh, Head of Global Medical Affairs Core Products at Sobi.

Orfadin is approved in the US and several other countries for the treatment of patients with confirmed diagnosis of hereditary tyrosinaemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine. Orfadin, together with the appropriate diet, is an essential part of effective HT-1 treatment. In April 2016, Sobi announced the approval of the Orfadin oral suspension in the US, thereby providing the possibility to now personalise treatments with five dosing alternatives: 2mg, 5mg, 10mg, 20mg capsules and 4mg/ml oral suspension.