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NITYR is a brand name for Nitisinone. The medicine, its uses, side effects and dosage are the same regardless of brand.
Used for: 1 INDICATIONS AND USAGE NITYR ® is 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. NITYR is a hydroxyphenyl-pyruvate dioxygenase inhibitor indicated for the treatment of adult and pediatric…
Verbatim from this product's FDA label. Tap a section to expand.
5 mg/kg (actual body weight) administered orally twice daily. 1 ) Administer NITYR with or without food. 1 ) Maintain dietary restriction of tyrosine and phenylalanine when administering NITYR. 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.
1 ) See the full prescribing information for dosage titration and monitoring. 2 ) See the full prescribing information for preparation and administration instructions. 5 mg/kg (actual body weight) administered orally twice daily. Titrate the dose in each individual patient based on biochemical and/or clinical response.
3 )] . Maintain dietary restriction of tyrosine and phenylalanine when administering NITYR. 2 )] . A maximum total daily dosage of 2 mg/kg may be needed based on the evaluation of all biochemical parameters. Missed Dose If a dose of NITYR is missed, do not administer two doses at once to make up for a missed dose.
Take the next dose at the scheduled time. 2 Dosage Titration and Monitoring Monitor plasma and/or urine succinylacetone concentrations, liver function parameters and alpha-fetoprotein levels. e. plasma and/or urine succinylacetone, urine 5-aminolevulinate (ALA), and erythrocyte porphobilinogen (PBG)-synthase activity during initiation of therapy, when switching from twice daily to once daily dosing, or if there is a deterioration in the patient’s condition.
75 mg/kg twice daily. If the biochemical response is satisfactory (undetectable blood and/or urine succinylacetone), the dosage should be adjusted only according to body weight gain and not according to plasma tyrosine levels. 1 )] .
In patients who develop plasma tyrosine levels above 500 micromol/L, assess dietary tyrosine and phenylalanine intake. Do not adjust the NITYR dosage in order to lower the plasma tyrosine concentration. 3 Preparation and Administration Instructions For patients who have difficulty swallowing intact tablets, disintegrate NITYR in water and administer using an oral syringe.
For patients who can swallow semi-solid foods, NITYR tablets can be crushed and mixed with applesauce. Administration of NITYR with other liquids or foods has not been studied and is not recommended.
6 ADVERSE REACTIONS 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.
1 ) To report SUSPECTED ADVERSE REACTIONS, contact Cycle Pharmaceuticals Ltd. gov/medwatch. 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 enrollment (median age 9 months), who were diagnosed with HT-1 by the presence of succinylacetone in the urine or plasma.
5 mg/kg twice daily, and the dose was increased in some patients to 1 mg/kg twice daily based on weight, biochemical, and enzyme markers. 1 ) ]. 1 to 80 months). 2 )] . Fourteen patients experienced ocular/visual events. The duration of the symptoms varied from 5 days to 2 years.
Six patients had thrombocytopenia, three of which had platelet counts 30,000/microL or lower. In 4 patients with thrombocytopenia, platelet counts gradually returned to normal (duration up to 47 days) without change in the nitisinone dose.
No patients developed infections or bleeding as a result of the episodes of leukopenia and thrombocytopenia. Patients with HT-1 are at increased risk of developing porphyric crises, hepatic neoplasms, and liver failure requiring liver transplantation.
5 WARNINGS AND PRECAUTIONS 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.
1 ) Do not adjust NITYR dosage in order to lower the plasma tyrosine concentration. 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. 1 ) Leukopenia and Severe Thrombocytopenia: Monitor platelet and white blood cell counts. 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.
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.
4 CONTRAINDICATIONS None. None. ( 4 )
Not medical advice. Always read the patient information leaflet and follow your prescriber or pharmacist.
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Preparation and Administration of NITYR with Water in an Oral Syringe:
Do not prepare more than two tablets at once within the same oral syringe. If patient’s dosage requires more than two tablets, follow the steps below using multiple oral syringes and prescribed number of tablets to achieve the required dose.
One Tablet 1. Remove the plunger from the 5-mL oral syringe and insert a single, intact tablet. 2. 6 mL of room temperature water. 3. Cap the oral syringe and leave the oral syringe for at least the length of time below: • 15 minutes for a 2 mg tablet • 60 minutes for a 5 mg or 10 mg tablet 4.
Turn the oral syringe up and down for at least 30 seconds to suspend the material. 5. Inspect the syringe to ensure the tablet has fully disintegrated. If the tablet is not fully disintegrated, leave the oral syringe for an additional 10 minutes.
Do not administer unless the tablet has fully disintegrated. Before administration of the suspension, turn the oral syringe up and down for 30 seconds to ensure the particles are suspended. 6. Uncap the oral syringe and administer all the suspension into the patient's mouth.
To facilitate full administration, avoid depressing the plunger to the end of the oral syringe and leave a gap between the plunger and the oral syringe. 7. Fill the oral syringe by drawing up 2 mL of water. Shake well and administer while this time fully depressing the plunger.
If particles are still present in the syringe, repeat this step. Two Tablets 1. Remove the plunger from the 5-mL oral syringe and insert two intact tablets. 2. Replace the plunger and draw up 5 mL of room temperature water. 3. Cap the oral syringe and leave it for at least the length of time below: • 15 minutes for 2 mg tablets • 60 minutes for 5 mg or 10 mg tablets 4.
Turn the oral syringe up and down for at least 30 seconds to suspend the material. 5. Inspect the syringe to ensure the tablets have fully disintegrated. If the tablet is not fully disintegrated, leave the oral syringe for an additional 10 minutes.
Do not administer unless the tablet has fully disintegrated. Before administration of the suspension, turn the oral syringe up and down for 30 seconds to ensure the particles are suspended. 6. Uncap the oral syringe and administer all the suspension into the patient's mouth.
To facilitate full administration, avoid depressing the plunger to the end of the oral syringe and leave a gap between the plunger and the oral syringe. 7. Fill the oral syringe by drawing up 2 mL of water. Shake well and administer while this time fully depressing the plunger.
If particles are still present in the syringe, repeat this step. Storage Instructions for the NITYR with water in an Oral Syringe The suspension can be stored at room temperature in the capped oral syringe, protected from direct sunlight for up to 24 hours after adding water to the tablets.
Discard after 24 hours. Preparation and Administration of NITYR Mixed in Applesauce For patients who can swallow semi-solid food, NITYR can be crushed and mixed with applesauce. 1. , clean glass). 2. Crush only 1 tablet at a time between two teaspoons forming a fine powder.
Repeat this step if more than 1 tablet is needed. 3. Transfer the powder to the applesauce container ensuring all the powder is transferred and no powder residue remains on the teaspoon. 4. Mix the powder into the applesauce until the powder is well dispersed.
5. Administer the entire NITYR-applesauce mixture to the patient immediately or within 2 hours of mixing. 6. To ensure that there is not any remaining NITYR-applesauce mixture, add approximately one teaspoon of applesauce to the same container and administer to the patient.
Storage Instructions for NITYR Mixed in Apple Sauce The mixture can be stored at room temperature, out of direct sunlight, for up to 2 hours after adding the crushed tablets to the applesauce. Discard after 2 hours.
These complications of HT-1 were observed in patients treated with nitisinone for a median of 22 months during the clinical trial (liver transplantation 13%, liver failure 7%, malignant hepatic neoplasms 5%, benign hepatic neoplasms 3%, porphyria 1%).
The most common adverse reactions reported in the clinical trial are summarized in TABLE 1. *reported in at least 1% of patients; ** another oral formulation of nitisinone TABLE 1 Most Common Adverse Reactions * in Patients with HT-1 Treated with Nitisinone ** Elevated tyrosine levels >10% Leukopenia 3% Thrombocytopenia 3% Conjunctivitis 2% Corneal Opacity 2% Keratitis 2% Photophobia 2% Eye Pain 1% Blepharitis 1% Cataracts 1% Granulocytopenia 1% Epistaxis 1% Pruritus 1% Exfoliative Dermatitis 1% Dry Skin 1% Maculopapular Rash 1% Alopecia 1% Adverse reactions reported in less than 1% of the patients, included death, seizure, brain tumor, encephalopathy, hyperkinesia, cyanosis, abdominal pain, diarrhea, enanthema, gastrointestinal hemorrhage, melena, elevated hepatic enzymes, liver enlargement, hypoglycemia, septicemia, and bronchitis.
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. 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.