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.
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.
| Common Brands | Zokinvy |
|---|---|
| Drug Class | Farnesyltransferase inhibitor |
| Controlled Substance Classification | Not a controlled medication |
| Generic Status | No lower-cost generic available |
| Availability | Prescription only |
The following side effects may get better over time as your body gets used to the medication. Let your healthcare provider know immediately if you continue to experience these symptoms or if they worsen over time.
Contact your healthcare provider immediately if you experience any of the following.
Side effects that you should report to your care team as soon as possible:
Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):
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.
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.
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.
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.
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.
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).
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.
Contraindications are specific health conditions or situations in which a person should not take a medication due to safety concerns. If you have any of the following conditions or if any of the following apply to you, let your healthcare provider know because Zokinvy (lonafarnib) will not be safe for you to take.
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Progeria is a rare, life-threatening, genetic condition where your body has a buildup of progerin, a mutated protein. These proteins are toxic and can cause severe heart problems (e.g., stroke or heart attack) and bone problems (e.g., deformed bones). Over time, progeria can cause early aging and lead to death in young people. Zokinvy helps to lower the amount of progerin in people with progeria, providing relief from symptoms and helping them live longer.
Zokinvy is the first and currently only oral medication to reduce the risk of death in people with progeria. In studies, this medication has been shown to extend life about an average of 2.5 years longer in people with progeria. If you have any questions about Zokinvy, follow up with your provider for more information.
The most common side effects of Zokinvy are vomiting, nausea, and diarrhea. These side effects are most common in the first month of treatment, but they get better over time. To help relieve symptoms of nausea, make sure to take each dose of Zokinvy with food and a lot of water. To help with diarrhea, you might be able to take a lower dose of loperamide (Imodium A-D). But keep in mind that Zokinvy can worsen side effects of loperamide (Imodium A-D,) such as bloating and dizziness. Talk with your provider if your nausea, vomiting, or diarrhea don't get better after several months. Your provider can adjust your dose of Zokinvy or provide medications to help with your symptoms.
Zokinvy hasn’t been studied in pregnant people, but animal studies showed that this medication caused serious harm to baby animals (e.g., deformed bones) and mothers (e.g., severe weight loss and loss of pregnancy). If you're able to become pregnant, make sure to use birth control, like condoms, while taking Zokinvy. If you’re pregnant or planning on becoming pregnant, talk to your provider so you can discuss the risks of taking Zokinvy.
Zokinvy hasn’t been studied in human fertility (the ability to have children), but animal studies showed that this medication lowered the ability to have babies in both male and female rats. While animal studies don’t always represent what happens in humans, it’s possible that Zokinvy can lower your ability to have children. If you plan to have children, please talk with your provider about the benefits and risks of taking this medication.
