HT-1 is a rare genetic disease in infants and children. It could lead to liver and kidney complications and become fatal if not treated.
Orfadin is indicated as adjunct treatment to dietary restriction of tyrosine and phenylalanine. The higher capsule strength of 20 mg was developed to support the treatment regimens of adolescent and adult patients as dosing is weight-based.
The availability of the higher strength could enable patients to use fewer capsules.
Sobi global medical affairs head Michael Yeh Core Products "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."
Orfadin blocks the breakdown of tyrosine amino acid in order to reduce the amount of toxic tyrosine by-products in the body.
It is approved in the US and various other countries to treat patients with confirmed diagnosis of HT-1 in combination with dietary restriction of tyrosine and phenylalanine.
Orfadin capsules are already available as 2mg, 5mg, and 10mg strengths. It was also approved as a 4mg/mL strength oral suspension in April this year.
Image: Orfadin is indicated as adjunct treatment to dietary restriction of tyrosine and phenylalanine. Photo: courtesy of Swedish Orphan Biovitrum AB.