Lasinone (Nitisinone) for HT-1 Treatment

What is Lasinone?

Lasinone is an enzyme inhibitor used to treat rare inherited metabolic disorders, specifically hereditary tyrosinemia type-1 a rare disorder that occurs due to abnormal accumulation of tyrosine, a protein.

How Lasinone Works?

Hereditary tyrosinemia is a rare disorder in which there is abnormal build-up of an amino acid called tyrosine in various organs like liver, kidney, or the nervous system. The signs and symptoms may include eye pain and redness, excessively watery eyes, abnormal light sensitivity (photophobia), or thick, painful skin on the palms and soles. Nitisinone works by blocking the enzyme 4-hydroxyphenylpyruvate dioxygenase, which is part of the body's process for breaking down the amino acid tyrosine.

Who Should Take Lasinone?

Lasinone is indicated for individuals with rare genetic disorders: hereditary tyrosinemia type 1 (HT-1) in this conditions the body cannot properly break down the amino acids tyrosine and phenylalanine. Treatment with Nitisinone requires a strict, lifelong diet restricted in these amino acids.

Product Specifications

Dosage and Administration:

• The recommended starting dosage is 0.5 mg/kg (actual body weight) administered orally twice daily.
• The recommended maintenance dosage of Lasinone 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.

Administration Guidelines:

• Administer Lasinone with or without food.
• Maintain dietary restriction of tyrosine and phenylalanine when administering Lasinone.

Monitoring Requirements

Biochemical monitoring

• Succinylacetone (SA): This is the primary marker for metabolic control. It should be undetectable in the blood and urine, which is the main goal of treatment.
• Nitisinone: Monitor blood Nitisinone levels to ensure they are within the therapeutic range (typically 40–60 µmol/L) to achieve more than 99% inhibition of p-HPPD activity. Some patients may require higher levels to completely suppress SA excretion.
• Tyrosine: Monitor plasma tyrosine levels to ensure they remain below 500 µmol/L to prevent toxicity. However, the Nitisinone dosage should not be altered solely to change tyrosine levels.

Clinical and other monitoring

• Liver function: Regularly check liver function markers.
• Alpha-fetoprotein (AFP): Monitor AFP levels, particularly to screen for hepatocellular carcinoma (HCC).
• Neurocognitive development: Conduct regular neurocognitive assessments, as some patients may develop neurocognitive deficits despite treatment.
• Diet: Assess the patient’s dietary intake of phenylalanine and tyrosine and ensure they are following the prescribed low-protein diet with a special metabolic formula.

Dose adjustments

• If succinylacetone is detectable after four weeks, the Nitisinone dose should be increased.
• Adjustments may be needed based on body weight gain.

Important Considerations

• Treatment must be continuous to avoid serious complications like acute liver failure or neurological crises.
• Early diagnosis and treatment, ideally through new-born screening, lead to significantly better outcomes and reduced risk of HCC.
• Certain side effects like photophobia have been reported but can often be managed with stricter dietary control.

Common 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.

Serious Warnings

• Ocular Symptoms, Developmental Delay and Hyperkeratotic Plaques: Due to elevated plasma tyrosine.
• Do not adjust Lasinone dosage in order to lower the plasma tyrosine concentration.
• Leukopenia and Severe Thrombocytopenia: Monitor platelet and white blood cell counts.

Drug 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.
• OAT1/OAT3 Substrates: Increased systemic exposure of these co-administered drugs; monitor for potential adverse reactions.

Storage Requirements

Store at room temperature between 15–30°C (59–86°F).

Product Pricing and Availability

• Available Pack Sizes: 60 Capsules per bottle.
• MRP: Lasinone 2 mg – ₹28,124; Lasinone 5 mg – ₹37,499; Lasinone 10 mg – ₹46,874.
• Where to Purchase: Call customer care number: 7337585050.
• Last Updated: November 27, 2025

Frequently Asked Questions (FAQ)

FDA Approval and Clinical Evidence

Clinical evidence

Effect of Nitisinone (NTBC) treatment on the clinical course of hepatorenal tyrosinemia

• Researcher recorded the clinical course of 78 Québec HT1 patients born between 1984 and 2004. There were three groups: those who never received Nitisinone (28 patients), those who were first treated after 1 month of age (26 patients) and those treated before 1 month (24 patients). Retrospective chart review was performed for events before 1994, when Nitisinone treatment began, and prospective data collection thereafter.

Key Findings:

• No hospitalizations for acute complications of HT1 occurred during 5731 months of Nitisinone treatment, versus 184 during 1312 months without treatment (p < 0.001).
• Liver transplantation was performed in 20 non-Nitisinone-treated patients (71%) at a median age of 26 months, versus 7 late-treated patients (26%, p < 0.001), and no early-treated patient (p < 0.001).
• No early-treated patient has developed detectable liver disease after more than 5 years.
• Ten deaths occurred in non-Nitisinone treated patients versus two in treated patients (p < 0.01). Both of the latter deaths were from complications of transplantation unrelated to HT1.

Conclusion:

Nitisinone treatment abolishes the acute complications of HT1. Some patients with established liver disease before Nitisinone treatment eventually require hepatic transplantation. Patients who receive Nitisinone treatment before 1 month had no detectable liver disease after more than 5 years.

Long-term safety and outcomes in hereditary tyrosinemia type 1 with Nitisinone treatment: a 15-year non-interventional, multicentre study

Study Type: 15-year non-interventional, non-comparative, multicentre study

Countries: 17 across Europe study

Patients: 315 patients were enrolled during the study period (complete set) 339 in extended analysis.

Patients diagnosed by neonatal screening started Nitisinone treatment at a median age of 0.8 months, compared to 8.5 months for those diagnosed clinically. Patients were followed-up with an investigator at least annually for as long as they were treated, or until the end of the study.

Age at start of Nitisinone treatment <28 days, ≥28 days to <6 months, ≥6 months to <12 months, and ≥12 months.

Standard dose: The average daily Nitisinone dose decreased over time, being highest in infants (1.20 mg/kg) and lowest in adults (0.79 mg/kg).

Key Outcome:

• Early initiation (<28 days):
• 0 deaths or liver transplants
• Lowest incidence of hepatic complications
• Median treatment duration: 11.2 years
• Good/Very good clinical condition in 87–98% of patients throughout 1 year of treatment
• No new safety signals identified

Conclusion:

Long-term Nitisinone treatment was well tolerated and no new safety signals were revealed. Life-limiting hepatic disease appears to have been prevented by early treatment start. Neonatal screening was the most effective way of ensuring early treatment. Standardised monitoring of blood tyrosine, phenylalanine, and Nitisinone levels has potential to guide individualised therapy.

Patient Support and Resources

Important Reminders

• Take Lasinone exactly as prescribed by your healthcare provider
• Do not adjust your dose without medical supervision
• Keep all scheduled appointments for monitoring
• Report any new symptoms or concerns promptly
• Store medication properly and check expiration dates
• Ensure adequate supply and refill prescriptions in advance
• Educate family members and caregivers about your condition and emergency signs