FDA Approves Oral Suspension Formulation for HT-1 Treatment

The Food and Drug Administration (FDA) has approved Orfadin (nitisinone; Sobi) Oral Suspension for the treatment of hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine. 

Orfadin is currently the only FDA-approved therapy for the treatment of HT-1. The new oral suspension formulation provides an alternate option to the capsules for infants, small children, and anyone with swallowing difficulties. 

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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 Oral Suspension will be available as a 4mg/mL strength in 100mL bottles containing 90mL of suspension. Orfadin is already available as 2mg, 5mg, and 10mg strength capsules in 60-count bottles. The oral suspension is anticipated to launch in August 2016. 

For more information call (866) 773-5274 or visit Orfadin.com.

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