Nitisinone is primarily used in the treatment of hereditary tyrosinemia type 1, a rare genetic disorder that affects the metabolism of the amino acid tyrosine. By inhibiting an enzyme involved in the tyrosine breakdown pathway, it prevents the accumulation of toxic metabolites that can cause severe liver and kidney damage. This medication helps manage symptoms, improve liver function, and reduce the risk of liver failure in affected patients. Additionally, nitisinone is sometimes used in the management of alkaptonuria, another genetic disorder, where it slows the progression of associated symptoms by reducing the production of harmful byproducts. Its application significantly improves the quality of life for individuals with these conditions.