Zokinvy
Zokinvy (lonafarnib) is an oral medication that's used for two rare, genetic conditions called progeria (or Hutchinson-Gilford progeria syndrome) and certain processing-deficient progeroid laminopathies (PDPL). It needs to be taken twice a day with meals. Zokinvy (lonafarnib) is only available at specialty pharmacies, so your child’s provider or pharmacist will help you set that up.
What is Zokinvy (lonafarnib)?
What is Zokinvy (lonafarnib) used for?
Lowering the risk of death in people with progeria (or Hutchinson-Gilford progeria syndrome)
Treatment of processing-deficient progeroid laminopathies (PDPL) in certain people
How Zokinvy (lonafarnib) works
Zokinvy (lonafarnib) is a farnesyltransferase inhibitor. It blocks an important process in people with progeria or PDPL by preventing buildup of harmful progerin and progerin-like proteins. Progerin is a mutated protein that causes cell damage. People with progeria or PDPL often have too much progerin. This can lead to early aging, as well as bone and heart problems. Zokinvy (lonafarnib) helps to lower the amount of these harmful proteins, which then lowers the risk of heart damage and extends the life in these young adults and children.
Drug Facts
What are the side effects of Zokinvy (lonafarnib)?
Common Side Effects
- Vomiting (90%)
- Diarrhea (81%)
- Nausea (56%)
- Loss of appetite (53%)
- Tiredness (51%)
- Upper respiratory tract infection (e.g., sore throat, runny or stuffy nose, postnasal drip; 51%)
- Belly pain (48%)
- Muscle or joint pain (48%)
- Weight loss (37%)
- Headache (37%)
- Cough (33%)
Other Side Effects
- Constipation
- Passing gas
- Dehydration
- Fever
- Vision changes, pink eye, or teary eyes
- Mouth sores
- Rash
- Itchy skin
- Depression
Serious Side Effects
- Brain ischemia (not enough blood flow to the brain): feeling weak on one side of the body, feeling weak in the arm or leg, confusion, trouble speaking, sudden vision changes, dizziness, loss of balance, severe headache
- Infections: fever, belly pain, diarrhea, cough, chills, sore throat, runny or stuffy nose, body aches
Source: DailyMed
The following side effects have also been reported
Side effects that you should report to your care team as soon as possible:
- Allergic reactions—skin rash, itching, hives, swelling of the face, lips, tongue, or throat
- Blurry vision, vision loss, seeing wavy or bent objects or blind spots with dark, light, or flashing spots
- High calcium level—increased thirst or amount of urine, nausea, vomiting, confusion, unusual weakness or fatigue, bone pain
- High potassium level—muscle weakness, fast or irregular heartbeat
- Infection—fever, chills, cough, or sore throat
- Kidney injury—decrease in the amount of urine, swelling of the ankles, hands, or feet
- Low potassium level—muscle pain or cramps, unusual weakness or fatigue, fast or irregular heartbeat, constipation
- Low red blood cell level—unusual weakness or fatigue, dizziness, headache, trouble breathing
- Low sodium level—muscle weakness, fatigue, dizziness, headache, confusion
- Unusual bruising or bleeding
Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):
- Cough
- Diarrhea
- Headache
- Muscle pain
- Nausea
- Vomiting
- Weight loss
Pros and cons of Zokinvy (lonafarnib)
Pros
The first and only oral medication for progeria and PDPL
Can be mixed with liquid sweetener, applesauce, or orange juice, so it's easy to take for people who have difficulty swallowing pills
Shown to extend life about 2.5 years longer in people with progeria
Cons
Only available as a brand medication, so it might be expensive
More likely to cause severe kidney problems
Interacts with many medications, such as atorvastatin (Lipitor), digoxin (Lanoxin), and diltiazem (Cardizem)
Pharmacist tips for Zokinvy (lonafarnib)
Take Zokinvy (lonafarnib) with food to lower the risk of vomiting, nausea, or diarrhea.
If you or your child prefers to swallow capsules, swallow the Zokinvy (lonafarnib) capsule whole with a lot of water. Don't chew the capsule.
If you or your child can’t swallow capsules, open up the Zokinvy (lonafarnib) capsule and mix each capsule’s content with 5 to 10 ml of a sweetened oral liquid, such as Ora Blend SF or Ora-Plus. If you can't find these sweeteners, you can also mix the contents with 1 or 2 teaspoonfuls (5 or 10 ml) of applesauce or orange juice (not made with Seville oranges). Take the entire mixture by mouth within 10 minutes.
Don't drink juices containing grapefruit or Seville oranges (also called bitter or sour oranges) during treatment with Zokinvy (lonafarnib). Doing so can cause the body to absorb more of this medication, causing greater risk of side effects.
Zokinvy (lonafarnib) can interact with many medications. Let your provider or pharmacist know what medications you're taking, including over-the-counter products and supplements, before you start Zokinvy (lonafarnib). Talk with your provider or pharmacist if you have any questions about interactions with this medication.
Tell your provider if you or your child experiences any changes in weight while taking Zokinvy (lonafarnib) because the dose might need to be changed. Let your provider know if you or your child has nausea, vomiting, or diarrhea that won’t go away since this can also be a cause of weight loss.
Try to take Zokinvy (lonafarnib) around the same times every day. If you accidentally forgot to take the medication, take your missed dose as soon as possible if you still have more than 8 hours until your next dose. But if it’s less than 8 hours until your next scheduled dose, then skip the missed dose and take the medication at your next regularly scheduled dose.
Frequently asked questions about Zokinvy (lonafarnib)
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What are the risks and warnings for Zokinvy (lonafarnib)?
Zokinvy (lonafarnib) can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.
Abnormal blood tests
Taking Zokinvy (lonafarnib) can cause abnormal blood tests, such as changes in electrolytes, lower blood cell counts, and higher levels of liver enzymes. These changes often get better as you continue treatment with this medication. Your provider will also do blood tests to monitor for any problems while you're using this medication.
High blood pressure
Zokinvy (lonafarnib) can sometimes raise your blood pressure. Your provider will check your blood pressure during treatment with this medication to make sure it doesn’t go too high and cause more problems. Talk to a provider right away if you have symptoms of high blood pressure, such as severe headaches, shortness of breath, unusual tiredness, dizziness, vision changes, irregular heartbeat, or chest pain.
Kidney problems
Risk factor: History of kidney problems
Zokinvy (lonafarnib) hasn’t been well studied in people with kidney problems, but animal studies showed that it caused serious kidney problems, such as kidney injury and changes in electrolytes. If you’re concerned about the potential for kidney damage, talk with your provider so they can decide how you can safely take Zokinvy (lonafarnib). You might need to get blood and urine tests done while you take Zokinvy (lonafarnib) so your provider can check your kidney health.
Vision problems
Risk factor: History of eye problems
Zokinvy (lonafarnib) hasn’t been well studied in people, but animal studies showed that it caused worsening night vision. You might need to get regular eye exams during treatment so your provider can make sure this medication is safe for you. If you experience cloudy or blurry vision, seeing halos around lights, or trouble seeing at night while taking Zokinvy (lonafarnib), please talk with your provider right away.
Interaction with other medications
Some medications can interfere with how your body gets rid of Zokinvy (lonafarnib). This can affect the amount of Zokinvy (lonafarnib) in your body, how well it works, and the risk of side effects.
Taking medications like ketoconazole and itraconazole (Sporanox) can raise the amount of Zokinvy (lonafarnib) in your body. This should be avoided since it can cause a higher risk of side effects.
Taking certain medications like rifampin should also be avoided. This is because rifampin can lower the amount of Zokinvy (lonafarnib) in your body and cause the medication to not work as well.
Zokinvy (lonafarnib) also interferes with how your body gets rid of other medications. For example, it shouldn’t be taken with some statins (e.g., lovastatin (Altoprev), simvastatin (Zocor), atorvastatin (Lipitor)). Doing so causes your body to absorb more of these statins and raise your risk for serious muscle problems. Zokinvy (lonafarnib) shouldn’t be taken together with midazolam (Versed) because it can cause severe dizziness, sleepiness, and slowed or difficult breathing.
Let your provider or pharmacist know what other medications you’re taking before and while taking Zokinvy (lonafarnib).
Zokinvy (lonafarnib) dosage forms
Typical dosing for Zokinvy (lonafarnib)
The dose of Zokinvy (lonafarnib) depends on body surface area (BSA), which is your body size calculated based on weight and height. Your provider will determine the number of capsules you or your child needs to take.
The usual starting dose is 115 mg/m2 by mouth twice daily with morning and evening meals. After 4 months, your provider might raise the dose to 150 mg/m2 twice daily with meals.
Zokinvy (lonafarnib) contraindications
Taking certain medications that affect how well Zokinvy (lonafarnib) is cleared from the body
Taking midazolam
Taking lovastatin, simvastatin, or atorvastatin
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