Nitysinone (Nitisinonum)

Pharmacodynamics Pharmacokinetics Indications Carefully Contraindications Dosing and Administration Side effects
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Clinical and pharmacological group: & nbsp

Other Metabolites

Included in the formulation
  • Orfadine
    capsules inwards 
    Svedish Orphan International AB     Sweden
    • Included in the list (Order of the Government of the Russian Federation No. 2782-r of 30.12.2014):

    VED

    АТХ:

    A.16.A.X   Other drugs for the treatment of digestive and metabolic diseases

    A.16.A.X.04   Nitysinone

    Pharmacodynamics:

    Competitively inhibits the enzyme, the precursor of fumarylacetoacetate hydrolase, 4-hydroxyphenyl-pyruvate dioxygenase in the pathway of tyrosine catabolism. Prevents the cumulation of maleicetatoacetate and fumarilacetoacetate, which in the body of patients are converted into succinylacetone and succinylacetoacetate, which have non-patho- and nephrotoxicity. In addition, succinylacetone inhibits the synthesis of porphyrin, which leads to the cumulation of 5-ammonulevulinate - a neurotoxin that causes porphyrin crises in hereditary tyrosinemia I type.

    Inhibition of tyrosine catabolism leads to an increase in the concentration of tyrosine in the blood plasma, so patients with hereditary tyrosinemia I type it is recommended to limit products containing phenylalanine and tyrosine.

    Pharmacokinetics:

    After oral administration, the empty stomach is completely absorbed in the gastrointestinal tract. The maximum concentration in blood plasma is reached after 3 hours.

    The elimination half-life is 54 hours. Elimination by the kidneys.
    Indications:

    It is used for the treatment of hereditary tyrosinemia I type with a diet with restriction of products containing phenylalanine and tyrosine.

    ICD-10

    IV.E70-E90.E70.2   Violations of tyrosine metabolism

    Contraindications:

    Individual intolerance.

    Carefully:

    Severe hepatic insufficiency.

    Pregnancy and lactation:

    Recommendations for FDA - Category B. It is used during pregnancy in those cases when the expected effect exceeds the negative consequences. Contraindicated in lactation.

    Dosing and Administration:

    Inside 1 mg / kg / day 2 times a day: morning and evening.

    The highest daily dose: 2 mg / kg / day.

    The highest single dose: 1 mg / kg / day.

    Side effects:

    Central and peripheral nervous system: headache, encephalopathy, hyperkinesia.

    Respiratory system: bronchitis, respiratory failure.

    The system of hematopoiesis: leukopenia, porphyria, thrombocytopenia, granulocytopenia.

    Digestive system: hepatic insufficiency, liver tumor, thirst, dehydration.

    Musculoskeletal system: pathological fractures.

    Dermatological reactions: maculopustular rash

    Reproductive system: amenorrhea.

    Sense organs: otitis, blepharokeratitis, cataract.

    Allergic reactions.

    Overdose:

    Increased side effects.

    Treatment is symptomatic.

    Interaction:

    No studies have been conducted.

    Special instructions:

    Monitoring of the functions of the liver, eyes, blood formulas.

    Instructions
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