Pronunciation:

loe-na-FAR-nib

Brand Names:

• Zokinvy

Dosage Forms:

• Capsule

Classifications:

Therapeutic—

Endocrine-Metabolic Agent

Uses of This Medicine:

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.

This medicine is available only with your doctor's prescription.

Before Using This Medicine:

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 this medicine, the following should be considered:

Allergies—

Tell your doctor if you have ever had any unusual or allergic reaction to this medicine 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.

Children—

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.

Older adults—

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

Breast-feeding—

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.

Other 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. When you are taking this medicine, it is especially important that your healthcare professional know if you are taking any of the medicines listed below. The following interactions have been selected on the basis of their potential significance and are not necessarily all-inclusive.

Using this medicine with any of the following medicines is not recommended. Your doctor may decide not to treat you with this medication or change some of the other medicines you take.

• Colchicine
• Gepirone
• Mavacamten

Using this medicine with any of the following medicines is usually not recommended, but may be required in some cases. If both medicines are prescribed together, your doctor may change the dose or how often you use one or both of the medicines.

• Aprepitant
• Aripiprazole
• Bosutinib
• Buprenorphine
• Dabrafenib
• Elexacaftor
• Fedratinib
• Fluticasone
• Fosaprepitant
• Ivacaftor
• Levoketoconazole
• Levomilnacipran
• Lurbinectedin
• Palovarotene
• Pimavanserin
• Pralsetinib
• Quizartinib
• Tezacaftor
• Valbenazine
• Vardenafil
• Vilanterol
• Vilazodone
• Zolpidem
• Zuranolone

Other interactions—

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 This Medicine:

Take this medicine 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 this medicine without first checking with your doctor.

This medicine 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 this medicine 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 this medicine. Do not mix this medicine with juice containing grapefruit or Seville oranges.

Dosing—

The dose of this medicine 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 this medicine. 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 this medicine, 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 This Medicine:

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

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

This medicine 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.

This medicine 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 this medicine have become infertile (unable to have children). Talk with your doctor before using this medicine 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.

Side Effects of This Medicine:

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.