Nitisinone Capsules

The FDA-approved generic medication to treat adult and pediatric patients with hereditary tyrosinemia type 1 (HT-1) in combination with dietary restrictions of tyrosine and phenylalanine

Important Safety Information

INDICATION
Nitisinone capsules are a hydroxy-phenylpyruvate dioxygenase inhibitor indicated for the treatment of adult and pediatric patients with hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine.

• Inadequate restriction of tyrosine and phenylalanine intake can lead to elevations in plasma tyrosine
• Maintain reduction in dietary tyrosine and phenylalanine while on nitisinone.
• Your healthcare provider should not adjust your dosage of nitisinone in order to lower the plasma tyrosine concentration.
• Plasma tyrosine levels greater than 500 micromol/L may lead to the following:
  • Ocular signs and symptoms including corneal ulcers, corneal opacities (cornea appears white or cloudy due to scarring), keratitis (inflammation of the cornea), conjunctivitis (pink eye - inflammation or infection of the outer membrane of the eye), eye pain, and light sensitivity
    • Your healthcare provider will obtain slit-lamp examination prior to treatment, regularly during treatment and may reexamine you if you develop symptoms or if your tyrosine levels exceed 500 micromol/L.
  • Variable degrees of intellectual disability and developmental delay
  • Painful hyperkeratotic plaques (thickening of the skin) on the soles and palms.
• Maintain plasma tyrosine levels below 500 micromol/L.

• You may develop leukopenia (low white blood cell count), severe thrombocytopenia (low number platelets, the cells that help the blood to clot), or both.
• Your healthcare provider will monitor white blood cell and platelet counts during treatment with nitisinone.

ADVERSE REACTIONS
The most common side effects (>1%) of nitisinone are elevated tyrosine levels, low platelet count, low white blood cell count, pink eye, white or cloudy cornea from scarring, inflammation of the cornea, light sensitivity, eye pain, inflammation of the eye lid, cataracts, low granulocytes - cells involved with immunity, nose bleeds, itching of the skin, redness and scaling of the skin, dry skin, rash and hair loss.

DRUG INTERACTIONS
Nitisinone can interfere with other medicines. Tell your healthcare provider about all the medicines you take.

• Pregnancy: Limited available data with nitisinone use in pregnant women are not sufficient to determine a drug-associated risk of adverse development outcomes. Tell your healthcare provider immediately if you are pregnant or plan to get pregnant.
• Lactation: There are no data on the presence of nitisinone in human milk, the effects on the breastfed infant, or the effects on milk production. Tell your healthcare provider immediately if you are breastfeeding or plan to breastfeed.
• Pediatric Use: The safety and effectiveness of nitisinone have been established in pediatric patients for the treatment of HT-1 in combination with dietary restriction of tyrosine and phenylalanine.
• Geriatric Use: Clinical studies of nitisinone did not include any subjects aged 65 and over.

Please read the Full Prescribing Information and Patient Instructions here

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088