Orfadin – The one and only FDA approved therapy for use as an adjunct to dietary restriction of tyrosine and phenylalanine for the treatment of hereditary tyrosinemia type 1 (HT-1)


Ordering Orfadin

Introducing Orfadin4U – your one source from ordering to treatment support –

Contact an Orfadin4U specialist at 877‐473‐3179 or via e‐mail at orfadin.us@remove-this.sobi.com.

 

Click here to download an order/enrollment form 

Indication and Usage

ORFADIN is a synthetic reversible inhibitor of 4-hydroxyphenylpyruvate dioxygenase indicated for use as an adjunct to dietary restriction of tyrosine and phenylalanine in the treatment of hereditary tyrosinemia type 1 (HT-1). 

Important Safety Information 

  • Inadequate restriction of tyrosine and phenylalanine intake can result in elevations in plasma tyrosine.
  • Do not adjust Orfadin dosage in order to lower the plasma tyrosine concentration.
  • Leucopenia and thrombocytopenia have been observed with treatment with Orfadin. Monitor platelet and white blood cell counts regularly during Orfadin therapy.
  • Most common adverse reactions (incidence >2%) are hepatic neoplasm, liver failure, thrombocytopenia, leucopenia, visual system complaints including conjunctivitis, corneal opacity, keratitis, and photophobia.
  • Use caution when administering ORFADIN with drugs that are metabolized by CYP2C9 because of a potential for increased systemic exposure of these drugs.
  • ORFADIN should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus. Based on animal data, ORFADIN may cause fetal harm.
  • Exercise caution when administering to a nursing woman. Because of the potential for serious adverse reactions in nursing infants from ORFADIN, a decision should be made to discontinue nursing or to discontinue the drug taking into account the importance of the drug to the mother. 

Please see full Prescribing Information