ORFADIN, with its active ingredient nitisinone, is a prescription medication primarily used in the treatment of hereditary tyrosinemia type 1 (HT-1), a rare genetic metabolic disorder. HT-1 is characterized by the body's inability to effectively break down the amino acid tyrosine, leading to the accumulation of toxic substances in the liver, kidneys, and central nervous system, which can cause serious health problems. ORFADIN works by inhibiting the enzyme 4-hydroxyphenylpyruvate dioxygenase, which is involved in the tyrosine degradation pathway. This inhibition reduces the formation of harmful substances, thereby preventing liver and kidney damage, and improving the quality of life for individuals with HT-1. ORFADIN is available in capsule and oral suspension forms and is typically part of a comprehensive treatment plan that includes dietary restrictions of tyrosine and phenylalanine.