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Limited coverage criteria – nitisinone

Last updated on March 19, 2025

 

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Generic name

nitisinone

Strength & form

2 mg/5 mg/10 mg/20 mg capsule

2 mg/5 mg/10 mg tablet

Special Authority criteria
for patients identified prenatally

Approval period

Initial and ongoing:

  • For the treatment of hereditary tyrosinemia type 1 (HT-1) in combination with a dietary restriction of tyrosine and phenylalanine, a prenatal diagnosis through genetic testing is required

Indefinite

Special Authority criteria
for patients identified through newborn screening

Approval period

Initial

For the treatment of hereditary tyrosinemia type 1 (HT-1) in combination with a dietary restriction of tyrosine and phenylalanine, a positive newborn screen for HT-1, specifically a blood succinylacetone measurement is required

Renewal

The HT-1 diagnosis is confirmed by:

A repeated blood succinylacetone measurement

OR

A urine succinylacetone measurement

Initial: 6 months

Renewal: Indefinite

Practitioner exemptions

  • None

Special notes

  • None

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