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Lonafarnib (Oral)

Medically reviewed by Drugs.com. Last updated on Oct 19, 2022.

Commonly used brand name(s)

In the U.S.

  • Zokinvy

Available Dosage Forms:

  • Capsule

Therapeutic Class: Endocrine-Metabolic Agent

Uses for lonafarnib

Lonafarnib is used to help reduce the risk of death in patients with Hutchinson-Gilford Progeria Syndrome (HGPS). HGPS is a genetic condition in which patients have a noticeable, rapid appearance of aging beginning in childhood.

Lonafarnib is also used to treat rare genetic disorders called processing-deficient Progeroid Laminopathies in patients with either heterozygous LMNA mutation with progerin-like protein accumulation, or homozygous or compound heterozygous ZMPSTE24 mutations.

Lonafarnib is available only with your doctor's prescription.

Before using lonafarnib

In deciding to use a medicine, the risks of taking the medicine must be weighed against the good it will do. This is a decision you and your doctor will make. For lonafarnib, the following should be considered:

Allergies

Tell your doctor if you have ever had any unusual or allergic reaction to lonafarnib or any other medicines. Also tell your health care professional if you have any other types of allergies, such as to foods, dyes, preservatives, or animals. For non-prescription products, read the label or package ingredients carefully.

Pediatric

Appropriate studies have not been performed on the relationship of age to the effects of lonafarnib in children younger than 12 months of age. Safety and efficacy have not been established.

Geriatric

Appropriate studies performed to date have not demonstrated geriatric-specific problems that would limit the usefulness of lonafarnib in the elderly.

Breastfeeding

There are no adequate studies in women for determining infant risk when using this medication during breastfeeding. Weigh the potential benefits against the potential risks before taking this medication while breastfeeding.

Interactions with medicines

Although certain medicines should not be used together at all, in other cases two different medicines may be used together even if an interaction might occur. In these cases, your doctor may want to change the dose, or other precautions may be necessary. Tell your healthcare professional if you are taking any other prescription or nonprescription (over-the-counter [OTC]) medicine.

Interactions with food/tobacco/alcohol

Certain medicines should not be used at or around the time of eating food or eating certain types of food since interactions may occur. Using alcohol or tobacco with certain medicines may also cause interactions to occur. Discuss with your healthcare professional the use of your medicine with food, alcohol, or tobacco.

Proper use of lonafarnib

Take lonafarnib exactly as directed by your doctor. Do not take it more often, and do not take it for a longer time than your doctor ordered. Do not stop taking lonafarnib without first checking with your doctor.

Lonafarnib usually comes with a patient information leaflet. Read and follow the instructions carefully. Ask your doctor or pharmacist if you have any questions.

Swallow the capsule whole with water. Do not chew it.

It is best to take lonafarnib with food.

If you cannot swallow the capsule whole, you may open it and mix the medicine with Ora Blend SF®, Ora-Plus®, orange juice, or applesauce. The mixture must be prepared fresh for each dose and taken within at least 10 minutes of mixing.

Do not eat grapefruit or Seville oranges while you are using lonafarnib. Do not mix lonafarnib with juice containing grapefruit or Seville oranges.

Dosing

The dose of lonafarnib will be different for different patients. Follow your doctor's orders or the directions on the label. The following information includes only the average doses of lonafarnib. If your dose is different, do not change it unless your doctor tells you to do so.

The amount of medicine that you take depends on the strength of the medicine. Also, the number of doses you take each day, the time allowed between doses, and the length of time you take the medicine depend on the medical problem for which you are using the medicine.

  • For oral dosage form (capsules):
    • For Hutchinson-Gilford Progeria Syndrome and processing-deficient Progeroid Laminopathies:
      • Adults and children 12 months of age and older with a body surface area (BSA) of 0.39 meter squared (m2)—At first, 115 milligrams per square meter (mg/m[2]), taken 2 times a day with the morning and evening meals. Your doctor may adjust your dose as needed and tolerated.
      • Children younger than 12 months of age—Use and dose must be determined by your doctor.

Missed dose

If you miss a dose of lonafarnib, take it as soon as possible. However, if it is almost time for your next dose, skip the missed dose and go back to your regular dosing schedule. Do not double doses.

If you miss a dose and it is 8 hours or more of your next scheduled dose, take it as soon as possible, and then go back to your regular schedule. If you miss a dose and it is less than 8 hours of your next scheduled dose, skip the missed dose and take your next dose at the regular time.

Storage

Store the medicine in a closed container at room temperature, away from heat, moisture, and direct light. Keep from freezing.

Keep out of the reach of children.

Do not keep outdated medicine or medicine no longer needed.

Ask your healthcare professional how you should dispose of any medicine you do not use.

Precautions while using lonafarnib

It is very important that your doctor check your progress at regular visits to make sure that lonafarnib is working properly. Blood and urine tests may be needed to check for unwanted effects.

Using lonafarnib while you are pregnant can harm your unborn baby. Use an effective form of birth control to keep from getting pregnant during treatment. If you think you have become pregnant while using the medicine, tell your doctor right away.

Do not use lonafarnib together with atorvastatin, ketoconazole, lovastatin, rifampin, or simvastatin. Do not use lonafarnib for 10 to 14 days before and 2 days after using midazolam.

Lonafarnib lowers the number of some types of blood cells in your body. Because of this, you may bleed or get infections more easily. To help with these problems, avoid being near people who are sick or have infections. Wash your hands often. Stay away from rough sports or other situations where you could be bruised, cut, or injured. Brush and floss your teeth gently. Be careful when using sharp objects, including razors and fingernail clippers.

Lonafarnib may cause kidney problems. Check with your doctor right away if you or your child have agitation, blood in the urine, coma, confusion, decreased urine output, depression, dizziness, headache, irritability, lethargy, muscle twitching, nausea, rapid weight gain, seizures, stupor, swelling of the face, ankles, or hands, or unusual tiredness or weakness.

Check with your doctor right away if eye pain or a change in vision occurs during treatment. These could be a sign of a serious eye problem. Your doctor may want your eyes be checked by an ophthalmologist (eye doctor).

Some men and women who use lonafarnib have become infertile (unable to have children). Talk with your doctor before using lonafarnib if you plan to have children.

Do not take other medicines unless they have been discussed with your doctor. This includes prescription or nonprescription (over-the-counter [OTC]) medicines and herbal or vitamin supplements.

Lonafarnib side effects

Along with its needed effects, a medicine may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.

Check with your doctor immediately if any of the following side effects occur:

More common

  • Agitation
  • black, tarry stools
  • blood in the urine or stools
  • burning, dry, or itching eyes
  • changes in the eye
  • coma
  • cough or hoarseness
  • confusion
  • dark urine
  • decreased appetite
  • decreased urine output
  • depression
  • diarrhea
  • discharge, excessive tearing
  • dizziness
  • fever
  • headache
  • irritability
  • lethargy
  • light-colored stools
  • lower back or side pain
  • muscle twitching
  • nausea
  • painful or difficult urination
  • pinpoint red spots on the skin
  • rapid weight gain
  • redness, pain, swelling of the eye, eyelid, or inner lining of the eyelid
  • seizures
  • sore throat
  • stomach pain or bloating
  • stupor
  • swelling of the face, ankles, or hands
  • unusual bleeding or bruising
  • unusual tiredness or weakness
  • vomiting
  • weight loss
  • yellow eyes or skin

Less common

  • Body aches or pain
  • chills
  • cough
  • difficulty in breathing
  • ear congestion
  • fever
  • headache
  • loss of voice
  • runny or stuffy nose
  • sneezing
  • sore throat

Some side effects may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects. Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:

More common

  • Bloated
  • bloody nose
  • constipation
  • excess air or gas in the stomach or bowels
  • full feeling
  • passing gas

Less common

  • Cracked lips
  • difficulty in swallowing
  • itching skin
  • sores, ulcers, or white spots in the lips, tongue, or inside the mouth

Other side effects not listed may also occur in some patients. If you notice any other effects, check with your healthcare professional.

Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.

Further information

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.