Orfadin (Nitisinone)

Orfadin (Nitisinone)

Orfadin

Nitisinone

Capsules in 2 mg, 5 mg, 10mg and 20 mg

Swedish Orphan Biovitrum

Medical Use

Orfadin (nitisinone) is a medication used to treat hereditary tyrosinemia type 1 (HT-1) in both adults and children. It works by inhibiting the enzyme hydroxy-phenylpyruvate dioxygenase, which prevents the buildup of harmful substances in the body. To effectively manage HT-1, Orfadin is usually prescribed along with a special diet that limits the intake of tyrosine and phenylalanine, essential amino acids. By lowering the levels of toxic substances, Orfadin helps prevent liver and kidney damage associated with HT-1, thereby enhancing the quality of life for patients with this rare genetic disorder.

Recommended Dosage:

  • -Starting Dosage: The initial recommended dosage of Orfadin is 0.5 mg/kg, taken orally twice a day.
  • -Maintenance Regimen: For patients aged 5 years and older who have undetectable levels of serum and urine succinylacetone after at least 4 weeks on a stable nitisinone dosage, the total daily dose of Orfadin can be administered once daily (e.g., 1 to 2 mg/kg once a day).