Medicines
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Discover all available medicines and treatments
Brand: Orfadin
Nitisinone is an orphan drug used to treat Hereditary Tyrosinemia type 1 (HT-1), a rare genetic disorder. It inhibits 4-hydroxyphenylpyruvate dioxygenase, an enzyme involved in tyrosine metabolism. By blocking this enzyme, nitisinone prevents the buildup of toxic metabolites that cause liver and kidney damage in HT-1 patients.
For the treatment of Hereditary Tyrosinemia type 1 (HT-1).
Outcome:
Increased risk of bleeding
Mechanism:
Nitisinone may interfere with vitamin K metabolism.
Outcome:
Increased risk of gastrointestinal bleeding
Mechanism:
Additive effects on platelet function.
Outcome:
Reduced nitisinone absorption
Mechanism:
Changes in stomach pH.
Most likely new formulation: Extended-release formulation (Year: 2026, 70% confidence)
Based on current clinical trial data and patient outcomes, continued approval for HT-1 treatment with high probability (95%).
Enzyme Inhibitor
Triketone