Nitisinone — uses, dosing, side effects & the brands that sell it · pharmaranks
Nitisinone: uses, dosing, side effects & brands
Nitisinone is a 4-hydroxyphenyl-pyruvate dioxygenase inhibitor sold in the U.S. under 3 brand and generic names, for tyrosinemias. Below: what the FDA label says, every product that contains it, what the pills look like, and its recall record.
By the pharmaranks editorial team·Reviewed against the FDA (openFDA label, NDC Directory & Enforcement) sources·How we research
Key facts
Drug class
4-Hydroxyphenyl-Pyruvate Dioxygenase Inhibitor
Treats
Tyrosinemias
Available as
Tablet · Capsule · Suspension
Sold as
3 products — Harliku, Nityr and Orfadin
Prescription?
Prescription only
Generic available?
Not in our catalog
How nitisinone is dosed
From the FDA label for Harliku (application NDA209449). Other nitisinone products — different forms, different strengths — are dosed differently. Follow the label for the one you were prescribed.
The recommended dosage of HARLIKU is 2 mg administered orally, once daily. ( 2.1 ) 2.1 Recommended Dosage The recommended dosage of HARLIKU is 2 mg administered orally, once daily. Administer HARLIKU with or without food [see Clinical Pharmacology ( 12.3 )] . Missed Dose If a dose of HARLIKU is missed, do not administer two doses at once to make up for a missed dose. Take the next dose at the scheduled time.
Nitisinone side effects
Most common adverse reactions (>1%) are elevated tyrosine levels, keratitis and thrombocytopenia. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Cycle Pharmaceuticals Ltd at 1-855-831-5413 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. Serious and/or clinically significant adverse reactions described elsewhere in labeling include: Ocular Symptoms and Hyperkeratotic Plaques Due to Elevated Plasma Tyrosine Levels [see Warnings and Precautions ( 5.1 )] Leukopenia and Severe Thrombocytopenia [see Warnings and Precautions ( 5.2 )] 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. The safety of HARLIKU was evaluated in Trial 1, a three-year, open-label, randomized, no-treatment controlled trial in 40 patients with AKU. Patients were between 38 and 68 years of age (27 male, 13 female) [see Clinical Studies ( 14 )] . Patients received either HARLIKU 2 mg orally once daily or no treatment [see Dosage and Administration ( 2 )]. The serious adverse reactions reported with HARLIKU were ocular/visual complaints associated with elevated tyrosine levels (keratitis) [see Warnings and Precautions ( 5.1 )] . Keratitis led to permanent…
Who shouldn’t take nitisinone
None. None. ( 4 )
Nitisinone drug interactions
Sensitive CYP2C9 Substrates: Reduce dosage of co-administered drug metabolized by CYP2C9 by half. ( 7.1 ) OAT1/OAT3 Substrates : Avoid concomitant use of HARLIKU with OAT1/OAT3 substrates. Concomitant use with OAT1/OAT3 substrates may increase the risk of adverse reactions related to the co-administered drug. ( 7.1 ) 7.1 Effects of HARLIKU on Other Drugs Sensitive CYP2C9 Substrates Reduce the dosage of the co-administered drug metabolized by CYP2C9 by half. Additional dosage adjustments may be needed to maintain therapeutic drug concentrations where minimal concentration changes may lead to serious adverse reactions. See prescribing information for those drugs. Nitisinone is a moderate CYP2C9 inhibitor. Nitisinone may increase exposure of co-administered drugs metabolized by CYP2C9 [see Clinical Pharmacology ( 12.3 )] . OAT1/OAT3 Substrates The concomitant use of HARLIKU with OAT1/OAT3 substrates may increase the risk of adverse reactions related to the co-administered drug. See prescribing information for those drugs. Nitisinone is an OAT1/OAT3 inhibitor which can lead to increased exposure of the co-administered drug. [ see Clinical Pharmacology ( 12.3 )] .
Every nitisinone product we track (3)
Same active ingredient — different manufacturer, form, price and FDA recall record. That last one is what our independent score measures.
Imprint codes, colour and shape from the FDA’s labelling data. Match the imprint on your pill — or search any imprint.
Nitisinone pill imprints
Imprint
Strength
Colour
Shape
Maker
L;2
2 mg
white
round
—
L;5
5 mg
white
round
—
L;10
10 mg
white
round
—
5;logo
5 mg
white
capsule
—
10;logo
10 mg
white
capsule
—
20;logo
20 mg
white
Frequently asked questions
What is nitisinone?
HARLIKU (nitisinone) is a hydroxyphenyl-pyruvate dioxygenase inhibitor. Nitisinone occurs as a white to yellowish-white, crystalline powder. It is practically insoluble in water, soluble in 2M sodium hydroxide and in methanol, and sparingly soluble in alcohol. The chemical name of nitisinone is 2-(2-nitro-4-trifluoromethylbenzoyl) cyclohexane-1,3-dione. The molecular formula is C 14 H 10 F 3 NO 5 and the molecular weight is 329.23. The structural formula is: Each HARLIKU (nitisinone) tablet contains 2 mg of nitisinone to be administered orally. The inactive ingredients are glyceryl dibehenate and lactose monohydrate. Figure 1 Structural Formula
What kind of drug is nitisinone?
The FDA classifies nitisinone as a 4-hydroxyphenyl-pyruvate dioxygenase inhibitor. If you are checking whether it is safe to combine with something else, the class is what matters — two drugs from the same class usually should not be stacked.
Can you take nitisinone with other medicines?
It depends on the medicine. We check it against the FDA labels rather than guessing: our interaction checker searches each drug's own label for the other and quotes what it says, naming the section it came from. Run nitisinone against whatever else you take — and remember that a label not naming a drug is not the same as that combination being safe.
What brand names is nitisinone sold under?
We track 3 nitisinone-containing products in the U.S.: Harliku, Nityr and Orfadin. They are the same active ingredient; they differ in form, manufacturer, price and FDA recall record.
What forms does nitisinone come in?
Across the brands we track, nitisinone is currently marketed as tablet, capsule and suspension, per the FDA's National Drug Code Directory. Each form is dosed differently — follow the label for the exact product you were prescribed.
Is there a generic nitisinone?
Sources: FDA openFDA drug label, National Drug Code Directory, and Enforcement (recall) database. This page reproduces public FDA data and is not medical advice. Dosing is set by your prescriber.
We do not currently list a generic-labelled nitisinone product. That does not always mean none exists — it means none appears under a generic name in the FDA data we track. Ask your pharmacist.