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)
Hereditary tyrosinemia type 1 (HT-1) is a rare, inherited metabolic disorder
caused by a deficiency
in the enzyme fumarylacetoacetate hydrolase (FAH), leading to a build-up of
the amino acid tyrosine
and its toxic by-products. This condition primarily affects the liver and
kidneys, and if untreated,
can lead to severe liver disease, liver failure, liver cancer, kidney
problems, and death.
Lasinone begins working very quickly to inhibit the toxic metabolic pathway
involved in hereditary
tyrosinemia type 1. While the toxic succinylacetone levels are suppressed
almost immediately, it can
take longer for other markers to return to normal. The median time for
plasma succinylacetone levels
to normalize was about 3.9 months in clinical studies.
No, Lasinone does not cure HT-1, but it is a life-saving treatment that
manages the disease by
preventing the build-up of toxic tyrosine metabolites. It blocks the
metabolic pathway that causes
the disease and significantly improves outcomes, preventing severe
complications like liver failure
and kidney dysfunction—especially when combined with a restricted diet of
tyrosine and phenylalanine.
Take the missed dose as soon as you remember, unless it is almost time for
your next scheduled dose.
Do not double doses to make up for a missed one.
Yes, the safety and effectiveness of Lasinone have been established for
treating HT-1 in combination
with dietary restriction of tyrosine and phenylalanine in pediatric
patients.
Yes, there are very strict dietary restrictions while taking Lasinone,
primarily involving a
low-protein diet that limits the amino acids tyrosine and phenylalanine.
These restrictions are
critical for preventing serious side effects. Foods to avoid include: Meat
(chicken, pork, lamb,
beef), Fish, Eggs, Dairy products (milk, cheese), Nuts, Seeds, Beans and
legumes.
No, pregnant or breastfeeding women should not take Lasinone, as it is not
recommended due to a lack
of data and potential risks to the infant.
For the treatment of hereditary tyrosinemia type 1 (HT-1), Lasinone is a
lifelong medication. The
condition is a permanent genetic disorder, and Lasinone works to manage the
symptoms rather than cure
the underlying disease.
During illness or stress, it is critical to do not stop taking Lasinone and
to adhere strictly to the
low-protein diet or your healthcare provider may suggest a specific
protocol. Contact your healthcare
provider immediately if you are sick, as illness can increase the risk of
complications.
When taking Lasinone, you will need regular laboratory tests to monitor
treatment effectiveness, check
for side effects, and ensure proper dosing. The specific tests and their
frequency will be determined
by your doctor, but they are crucial for managing your condition safely.
While Lasinone can be taken with other medications, it interacts with many
drugs and requires strict
medical supervision. You should tell your doctor and pharmacist about all
prescription,
over-the-counter, and herbal medications you are taking, as your doctor may
need to adjust the
dosages or monitor you closely for side effects.
Yes, genetic testing is available for family members of those with
hereditary type 1 tyrosinemia to
identify if they are carriers or affected by the condition. Molecular
genetic testing for the FAH gene
can confirm a diagnosis and is recommended to allow for genetic counselling
for family members.
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
Disclaimer
For more information about Lasinone or to discuss whether this medication is
right for you, consult with
your healthcare provider or geneticist specialist. Always read the complete
prescribing information
before starting any new medication.
This information is for educational purposes only and does not replace
professional medical advice.
Lasinone is a prescription medication that should only be used under medical
supervision.
References
For more clinical evidences refer below references:
-
Jean Larochelle et al. Effect of nitisinone (NTBC) treatment on the clinical
course of hepatorenal
tyrosinemia in Québec. Mol Genet Metab. 2012 Sep;107(1-2):49–54.
-
Ute Spiekerkoetter et al. Long-term safety and outcomes in hereditary
tyrosinaemia type 1 with
nitisinone treatment: a 15-year non-interventional, multicentre study. Lancet
Diabetes Endocrinol. 2021;
9: 427–35.
-
Anibh Martin Das et al. Clinical utility of nitisinone for the treatment of
hereditary tyrosinemia
type-1 (HT-1). The Application of Clinical Genetics. 2017;10:43–48.
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