nityr
Nityr (Nitisinone) is a 4-hydroxyphenyl-pyruvate dioxygenase inhibitor used to treat Tyrosinemias.
Nitisinone · by Cycle
Key facts
- Active ingredient
- Nitisinone
- Form
- Tablet
- Strength
- Nitisinone 10MG · Nitisinone 2MG · Nitisinone 5MG
- Type
- Prescription (Rx)
- Brand or generic
- Brand-name
- May treat
- tyrosinemias
- Manufacturer
- Cycle
- Half-life
- about 54 hours (mean terminal half-life) (how long it stays in your system)
- FDA application
- NDA209449
What is Nityr?
From the FDA label:NITYR (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 empirical formula is C 14 H 10 F 3 NO 5 and the molecular weight is 329.23. The structural formula is: Each NITYR (nitisinone) tablet contains 2, 5 or 10 mg of nitisinone. Inactive ingredients are: glyceryl dibehenate, and lactose monohydrate. NITYR tablets are intended for oral administration. Figure 1 Structural Formula
How to use
The recommended starting dosage is 0.5 mg/kg (actual body weight) administered orally twice daily. ( 2.1 ) Administer NITYR with or without food. ( 2.1 ) Maintain dietary restriction of tyrosine and phenylalanine when administering NITYR. ( 2.1 ) The recommended maintenance dosage of NITYR in patients 5 years of age and older who have undetectable serum and urine succinylacetone concentrations after a minimum of 4 weeks on a stable dosage of nitisinone, is 1 to 2 mg/kg once daily. ( 2.1 ) See the full prescribing information for dosage titration and monitoring. ( 2.2 ) See the full prescribing information for preparation and administration instructions. ( 2.2 ) 2.1 Recommended Dosage The recommended starting dosage of NITYR is 0.5 mg/kg (actual body weight) administered orally twice daily. Titrate the dose in each individual patient based on biochemical and/or clinical response. Administer NITYR with or without food [see Clinical Pharmacology ( 12.3 )] . Maintain dietary restriction of tyrosine and phenylalanine when administering NITYR. Maintenance Dosage The recommended maintenance dosage of NITYR in patients 5 years of age and older who have undetectable serum and urine succinylacetone concentrations after a minimum of 4 weeks on a stable dosage of nitisinone, is 1 to 2 mg/kg once daily [see Clinical Pharmacology ( 12.2 )] . A maximum total daily dosage of 2 mg/kg may be…
Side effects
Most common adverse reactions (>1%) are elevated tyrosine levels, thrombocytopenia, leukopenia, conjunctivitis, corneal opacity, keratitis, photophobia, eye pain, blepharitis, cataracts, granulocytopenia, epistaxis, pruritus, exfoliative dermatitis, dry skin, maculopapular rash and alopecia. ( 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, Developmental Delay 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 NITYR has been established based on studies of another oral formulation of nitisinone in patients with HT-1 [see Clinical Studies ( 14 )] . Below is a display of the adverse reactions of nitisinone in these studies. Nitisinone was studied in one open-label, uncontrolled study of 207 patients with HT-1, ages 0 to 22 years at…
Warnings
Important safety information
Ocular Symptoms, Developmental Delay and Hyperkeratotic Plaques Due To Elevated Plasma Tyrosine Levels: Inadequate restriction of tyrosine and phenylalanine intake can lead to elevations in plasma tyrosine and levels above 500 micromol/L may lead to ocular signs and symptoms, intellectual disability and developmental delay, or painful hyperkeratotic plaques on the soles and palms. ( 5.1 ) Do not adjust NITYR dosage in order to lower the plasma tyrosine concentration. ( 5.1 ) Obtain slit-lamp examination prior to treatment and regularly thereafter. Reexamine patients if symptoms develop or tyrosine levels are > 500 micromol/L. Assess plasma tyrosine levels in patients with an abrupt change in neurologic status. ( 5.1 ) Leukopenia and Severe Thrombocytopenia: Monitor platelet and white blood cell counts. ( 5.2 ) 5.1 Ocular Symptoms, Developmental Delay and Hyperkeratotic Plaques Due To Elevated Plasma Tyrosine Levels Treatment with NITYR may cause elevated plasma tyrosine levels in patients with HT-1. Inadequate restriction of tyrosine and phenylalanine intake can lead to elevations in plasma tyrosine levels and levels greater than 500 micromol/L may lead to the following: Ocular signs and symptoms including corneal ulcers, corneal opacities, keratitis, conjunctivitis, eye pain, and photophobia. All of which have been reported in patients treated with nitisinone [see Adverse Reactions ( 6.1 )] . In a clinical study in a non HT-1 population without dietary restriction and reported tyrosine levels > 500 micromol/L both symptomatic and asymptomatic keratopathies have been observed. Therefore, perform a baseline ophthalmologic examination including slit-lamp examination prior to initiating NITYR treatment and regularly thereafter. Patients who develop photophobia, eye pain, or signs of inflammation such as redness, swelling, or burning of the eyes or tyrosine levels are > 500 micromol/L during treatment with NITYR should undergo slit-lamp reexamination and immediate measurement of the plasma tyrosine concentration. Variable degrees of intellectual disability and developmental delay. In patients treated with NITYR who exhibit an abrupt change in neurologic status, perform a clinical laboratory assessment including plasma tyrosine levels. Painful hyperkeratotic plaques on the soles and palms. Maintain concomitant reduction in dietary tyrosine and phenylalanine while on NITYR treatment. In patients with HT-1 who are treated with NITYR and dietary restriction and develop elevated plasma tyrosine levels, assess dietary tyrosine and phenylalanine intake. Do not adjust NITYR dosage in order to lower the plasma tyrosine concentration. Maintain plasma tyrosine levels below 500 micromol/L. 5.2 Leukopenia and Severe Thrombocytopenia In clinical trials, patients treated with another oral formulation of nitisinone and dietary restriction developed reversible leukopenia (3%), thrombocytopenia (3%), or both (1.5%) [see Adverse Reactions ( 6.1 )] . No patients developed infections or bleeding as a result of the episodes of leukopenia and thrombocytopenia. Monitor platelet and white blood cell counts during NITYR therapy.
Who should not take Nityr
None. None. ( 4 )
Interactions
CYP2C9 Substrates : Increased systemic exposure of these co-administered drugs; reduce the dosage. Additional dosage adjustments may be needed to maintain therapeutic drug concentrations for narrow therapeutic index drugs. ( 7 ) OAT1/OAT3 Substrates : Increased systemic exposure of these co-administered drugs; monitor for potential adverse reactions. ( 7 ) Nitisinone is a moderate CYP2C9 inhibitor, a weak CYP2E1 inducer and an inhibitor of OAT1/OAT3. TABLE 2 includes drugs with clinically significant drug interactions when administered concomitantly with NITYR and instructions for preventing or managing them. TABLE 2. Clinically Significant Interactions Affecting Co-Administered Drugs Sensitive CYP2C9 Substrates (e.g., celecoxib, tolbutamide) or CYP2C9 Substrates with a Narrow Therapeutic Index (e.g., phenytoin, warfarin) Clinical Impact Increased exposure of the co-administered drugs metabolized by CYP2C9. [see Clinical Pharmacology ( 12.3 )] Prevention or Management Reduce the dosage of the co-administered drugs metabolized by CYP2C9 drug by half. Additional dosage adjustments may be needed to maintain therapeutic drug concentrations for narrow therapeutic index drugs. See prescribing information for those drugs. OAT1/OAT3 Substrates (e.g., adefovir, ganciclovir, methotrexate) Clinical Impact Increased exposure of the interacting drug. [see Clinical Pharmacology ( 12.3 )] Prevention or Management Monitor for potential adverse reactions related to the co-administered drug.
Storing Nityr, and how long it keeps
Quoted from this product’s own FDA label. Storage belongs to the product and its device, not to the drug in general — a pen and a tablet of the same medicine are kept completely differently.
- “Discard after 24 hours.”
- “Discard after 2 hours.”
- “Store NITYR tablets at room temperature between 20°C to 25°C (68°F to 77°F); excursions permitted between 15°C and 30°C (59°F and 86°F) [see USP Controlled Room Temperature].”
How long does Nitisinone stay in your body?
The elimination half-life of nitisinone is about 54 hours (mean terminal half-life) — the time it takes the body to clear about half of it. As a rule of thumb, a medicine is mostly gone after roughly 4 to 5 half-lives, though this varies from person to person with age and kidney or liver function. This is the terminal (elimination) half-life the ORFADIN label reports for a single dose in healthy male adults — roughly 2 days, which is why once-daily dosing works and why it takes about 2 weeks (steady state within 14 days) for levels to plateau after a change. The label does not describe an active metabolite, and nitisinone is not a prodrug: it is only minimally metabolized (in vitro, minor metabolism possibly via CYP3A4) and renal excretion is minor (about 3% of the dose unchanged in urine). The figure is formulation-specific: the NITYR tablet label reports a mean terminal half-life of 59.3 hours (SD 8.9) in healthy subjects. Populations: the label provides NO pharmacokinetic data in kidney impairment or liver impairment, and states that clinical studies included no subjects 65 or over and that no PK studies were done in geriatric patients (it only advises cautious dose selection in the elderly) — so how the half-life shifts in those groups is not established on the label.
This is general information, not medical advice. It doesn’t tell you when a drug test would read negative (tests detect substances for different, often longer, windows) or when it’s safe to take another dose — ask your pharmacist or prescriber. Source: ORFADIN (nitisinone) capsules/oral suspension — FDA label via DailyMed, §12.3 Pharmacokinetics (Elimination).
Drug class
May treat (source: NIH RxClass)
See how Nityr ranks — best-rated 4-hydroxyphenyl-pyruvate dioxygenase inhibitor for:
Dosage forms
Tablet
The forms currently marketed in the U.S., per the FDA National Drug Code Directory.
Ways to save
Ask for the generic
Same active ingredient, far cheaper. Is there a generic? →
Request a 90-day supply
Bulk fills usually lower the per-dose price vs monthly refills.
Use copay cards
Manufacturer copay cards & patient-assistance programs — especially for brand drugs.
Compare alternatives
A same-class option may cost less. See alternatives →
Frequently asked questions
- What does Nityr treat?
- Nityr (Nitisinone) may be used to treat tyrosinemias, based on NIH RxClass drug-classification data (conditions a drug may treat — not a verbatim copy of the FDA-approved label). This is general reference, not medical advice.
- Is there a coupon or discount for Nityr?
- pharmaranks is an independent ratings site, not a pharmacy — we don't issue coupons or sell Nityr. To pay less, ask your prescriber or pharmacist whether a generic equivalent exists, check the manufacturer's copay/savings card and patient-assistance program, and compare a pharmacy discount card against your insurance copay. Prices vary by pharmacy, insurance, and location, so always confirm at the counter. This is general reference, not medical or financial advice.
- Who makes Nityr?
- Nityr is marketed by Cycle. You can see Cycle's full profile, rating, and other products on pharmaranks.
- Is Nityr a brand-name or generic drug?
- Nityr is a brand-name product with the active ingredient Nitisinone.
- Is Nityr available over the counter?
- No. Nityr is a prescription (Rx) medication in the US — you need a prescription from a licensed clinician (in person or via telehealth); it isn't sold over the counter. For some conditions there are OTC alternatives — ask your pharmacist.
- What forms does Nityr come in?
- Nityr is currently marketed as tablet, per the FDA's National Drug Code Directory.
- What class of drug is Nityr?
- Nityr is classified as 4-hydroxyphenyl-pyruvate dioxygenase inhibitor, per the FDA's Established Pharmacologic Class.
- Is Nityr FDA-registered?
- Nityr is on record with the U.S. FDA under application number NDA209449. You can verify this on its official FDA label.
- Is Nityr safe?
- There's no single safe-or-not verdict, and we don't yet have a composite recall-safety score for Nityr. Review its FDA-label side effects and warnings below, and always consult a licensed professional.
- What are the side effects of Nityr?
- Nityr's side effects are taken directly from its FDA label. From the label: Most common adverse reactions (>1%) are elevated tyrosine levels, thrombocytopenia, leukopenia, conjunctivitis, corneal opacity, keratitis, photophobia, eye pain, blepharitis, cataracts, granulocytopenia, epistaxis, pruritus, exfoliative dermatitis, dry skin, maculopapular rash and alopecia.… Both common and serious reactions are documented in full on this page. This is general reference from the FDA, not medical advice — always consult a professional.
Clinical content sourced from the FDA label via openFDA (U.S. FDA). View the FDA label on DailyMed → Provided for general reference only — not medical advice. Always consult a licensed professional and the current prescribing information.
Reviews
No reviews yet. Be the first to write one.
People also viewed
Compare Nityr head-to-head
Browse medications A–Z
Research products from A to Z, compare independent ratings, and find alternatives.
nityr
New