Sobi announced that the Food and Drug Administration (FDA) has approved a new 20mg strength of Orfadin (nitisinone) capsules for the treatment of hereditary tyrosinemia type-1 (HT-1), a rare genetic disease in infants and children. 

Orfadin is indicated as adjunct treatment to dietary restriction of tyrosine and phenylalanine. The higher dosage strength was developed to support the treatment regimens of adolescents and adults as dosing is weight-based. The availability of the higher strength may allow patients to use fewer capsules. 

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Orfadin is a competitive 4-hydroxyphenyl-pyruvate dioxygenase inhibitor that works by blocking the normal catabolism of tyrosine, thus preventing the accumulation of catabolic intermediates maleylacetoacetate and fumarylacetoacetate. In patients with HT-1, these intermediates are converted to toxic metabolites that are responsible for hepatic and renal toxicity.

Orfadin capsules are already available as 2mg, 5mg, and 10mg strengths. Orfadin was also approved as a 4mg/mL strength oral suspension in April 2016. 

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