Lonafarnib
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Drug Status
Government Approvals
US(FDA), UK(BNF)
WHO Essential Medicine
NO
Known Teratogen
No
Pharmaceutical Class
Farnesyltransferase Inhibitor
Controlled Drug Substance
No
Lonafarnib
Summary
Lonafarnib is used to reduce the risk of death in patients with Hutchinson-Gilford Progeria Syndrome and certain progeroid laminopathies. These are rare genetic disorders that cause rapid aging in children.
Lonafarnib works by inhibiting an enzyme called farnesyltransferase. This prevents the buildup of abnormal proteins that cause rapid aging, helping to protect cells from damage and improve patient outcomes.
The usual starting dose of Lonafarnib for both adults and children 12 months of age and older is 115 mg/m, taken twice daily with meals. After 4 months, the dose is typically increased to 150 mg/m twice daily.
Common side effects of Lonafarnib include vomiting, diarrhea, nausea, decreased appetite, fatigue, and headache. Serious side effects can include heart arrhythmias and potential kidney and retinal toxicity.
Lonafarnib can interact with several drugs and should not be used with certain medications due to the risk of severe interactions. It can cause harm to an unborn baby, so pregnant women or women planning to become pregnant should not use it. It is also contraindicated in patients with severe liver impairment.
Indications and Purpose
How does Lonafarnib work?
Lonafarnib works by inhibiting an enzyme called farnesyltransferase, which is involved in the production of a protein that contributes to progeria symptoms. By blocking this enzyme, Lonafarnib helps reduce the buildup of defective proteins, improving health outcomes. Think of it like fixing a faulty assembly line to produce better products. This leads to increased lifespan and reduced cardiovascular complications.
Is Lonafarnib effective?
Lonafarnib is effective for treating certain genetic disorders, such as progeria, which is a rare condition causing rapid aging in children. Clinical studies show Lonafarnib improves health outcomes, including increased lifespan and reduced cardiovascular complications. Its effectiveness is supported by evidence from clinical trials and patient observations.
What is Lonafarnib?
Lonafarnib is a medication used to treat progeria, a rare genetic disorder causing rapid aging in children. It works by inhibiting a specific enzyme involved in the disease process. This helps improve health outcomes, such as increasing lifespan and reducing cardiovascular complications. Lonafarnib is used as a complementary addition to other therapies for managing progeria.
Directions for Use
For how long do I take Lonafarnib?
Lonafarnib is usually a long-term medication for managing conditions like progeria. You'll typically take it every day as a lifelong treatment unless your doctor suggests otherwise. How long you'll need this medication depends on your body's response and any side effects you experience. Always talk with your doctor before changing or stopping your Lonafarnib treatment.
How do I dispose of Lonafarnib?
Dispose of unused Lonafarnib through a drug take-back program or collection site at a pharmacy or hospital. If unavailable, mix the medication with something undesirable like used coffee grounds, seal it in a plastic bag, and throw it away in the trash. This helps prevent harm to people and the environment.
How do I take Lonafarnib?
Take Lonafarnib as directed by your doctor, usually twice daily with food. Swallow the capsules whole; do not crush or chew them. If you miss a dose, take it as soon as you remember unless it's almost time for your next dose. In that case, skip the missed dose and continue your regular schedule. Do not double up on doses. Follow any dietary or beverage restrictions your doctor advises while on this medication.
How long does it take for Lonafarnib to start working?
Lonafarnib begins working in your body shortly after you take it, but noticeable improvements in symptoms may take several weeks to months. The time it takes to achieve full therapeutic effects can vary based on individual factors like age and overall health. Regular check-ups with your doctor will help monitor your progress and adjust treatment as needed.
How should I store Lonafarnib?
Store Lonafarnib at room temperature, away from moisture and light. Keep it in a tightly closed container. Avoid storing it in humid places like bathrooms. Always keep Lonafarnib out of children's reach to prevent accidental ingestion. Check the expiration date regularly and dispose of any unused or expired medication properly.
What is the usual dose of Lonafarnib?
The usual starting dose of Lonafarnib for adults is 150 mg taken twice daily with food. Your doctor may adjust your dose based on your response and any side effects. The maximum recommended dose is 200 mg twice daily. Always follow your doctor's specific dosing instructions for your personal health needs. Special populations, such as children or the elderly, may require different dosing.
Warnings and Precautions
Can I take Lonafarnib with other prescription drugs?
Lonafarnib can interact with certain medications, increasing the risk of side effects or reducing effectiveness. It's important to inform your doctor about all medications you're taking. They can help manage potential interactions and adjust your treatment plan as needed to ensure safety and effectiveness.
Can Lonafarnib be taken safely while breastfeeding?
Lonafarnib is not recommended while breastfeeding. There's limited information on whether it passes into human breast milk. If you're taking Lonafarnib and want to breastfeed, talk with your doctor about safer medication options. They can help you make informed decisions to ensure your baby's safety.
Can Lonafarnib be taken safely while pregnant?
Lonafarnib is not recommended during pregnancy due to limited safety data. Animal studies suggest potential risks, but human data is lacking. If you're pregnant or planning to become pregnant, talk with your doctor about the safest treatment options. They can help create a plan that protects both you and your baby.
Does Lonafarnib have adverse effects?
Adverse effects are unwanted reactions to a medication. Common adverse effects of Lonafarnib include nausea, vomiting, and diarrhea. These effects are usually mild. Serious adverse effects, like liver problems, are rare but require immediate medical attention. Always inform your doctor about any new or worsening symptoms while taking Lonafarnib to ensure your treatment remains safe and effective.
Does Lonafarnib have any safety warnings?
Yes, Lonafarnib has important safety warnings. It can cause liver problems, so regular liver function tests are necessary. If you experience symptoms like yellowing of the skin or eyes, dark urine, or severe fatigue, contact your doctor immediately. Not adhering to these warnings can lead to serious health issues. Always follow your doctor's advice and report any unusual symptoms while taking Lonafarnib.
Is it safe to drink alcohol while taking Lonafarnib?
It's best to avoid alcohol while taking Lonafarnib. Alcohol can increase the risk of liver problems, which are a concern with Lonafarnib. If you choose to drink, limit your intake and watch for symptoms like nausea or jaundice, which is yellowing of the skin or eyes. Talk with your doctor about alcohol use while on this medication.
Is it safe to exercise while taking Lonafarnib?
You can exercise while taking Lonafarnib, but be cautious. This medication can cause dehydration, which might make you feel dizzy during exercise. Drink plenty of water before, during, and after physical activity. If you notice dizziness or unusual fatigue, slow down or stop exercising and rest. Consult your doctor if you have concerns.
Is it safe to stop Lonafarnib?
Stopping Lonafarnib suddenly can cause your condition to worsen. It's usually used long-term for chronic conditions. Always talk with your doctor before stopping Lonafarnib. They might suggest gradually reducing your dose or switching to a different medication to keep your condition under control. Your doctor will help you make any medication changes safely.
Is Lonafarnib addictive?
Lonafarnib is not addictive or habit-forming. This medication doesn't cause dependency or withdrawal symptoms when you stop taking it. Lonafarnib works by inhibiting a specific enzyme, which doesn't affect brain chemistry in ways that could lead to addiction. You won't experience cravings for this medication or feel compelled to take more than prescribed.
Is Lonafarnib safe for the elderly?
Elderly patients may be more vulnerable to side effects of Lonafarnib, such as liver problems. Regular monitoring and dose adjustments may be necessary. Discuss any concerns with your doctor, who can help ensure the medication is safe and effective for you.
What are the most common side effects of Lonafarnib?
Side effects are unwanted reactions that can happen when taking a medication. Common side effects of Lonafarnib include nausea, vomiting, and diarrhea. These occur in a small percentage of people. If you notice new symptoms after starting Lonafarnib, they might be temporary or unrelated to the medication. Talk with your doctor before stopping any medication.
Who should avoid taking Lonafarnib?
Don't take Lonafarnib if you're allergic to it or its ingredients. Serious allergic reactions require immediate medical help. Lonafarnib is contraindicated in patients with severe liver problems. Use caution if you have other health conditions, and always consult your doctor about these concerns. They will determine if Lonafarnib is safe for you.
