What is Zokinvy (lonafarnib)?
Zokinvy (zoh-KIN-vee) is a prescription medicine used to treat Hutchinson-Gilford Progeria Syndrome, a very rare genetic disease that causes children to age rapidly. Children with progeria appear to be healthy at birth but usually start to show signs of rapid aging in the first two years of their life. Symptoms, diagnosis and management are discussed.
Zokinvy, manufactured by Eiger Biopharmaceuticals is also used in the treatment of some types of Progeroid Laminopathies known as processing-deficient Progeroid Laminopathies. These illnesses are caused when the body makes harmful proteins called progerin and progerin-like proteins. The FDA approval of Zokinvy was based on results from the Observational Cohort Survival Study, which retrospectively compared survival data from two Phase 2 studies in patients with HGPS to those from a natural history cohort.
Zokinvy Mechanism of Action
Zokinvy (lonafarnib) inhibits farnesyltransferase to prevent farnesylation and subsequent accumulation of progerin and progerin-like proteins in the inner nuclear membrane.
The accumulation of defective, farnesylated proteins which form tight associations with the nuclear envelope, leading to cellular instability and the process of premature aging in children and young adults with Progeria and processing-deficient Progeroid Laminopathies.
How should I take Zokinvy?
Take Zokinvy exactly as your healthcare provider tells you to. Do not stop taking Zokinvy without talking to your healthcare provider. Your healthcare provider may change your dose of Zokinvy if needed.
Your healthcare provider will provide you with the dose you should be taking. The dose is based on body size (height and weight).
This medication is taken as described below:
• Start at 115 mg/m2 twice daily with morning and evening meals
• After 4 months, increase to 150 mg/m2 twice daily
• Round all total daily doses to nearest 25 mg increment
• Swallow capsules whole. If unable to swallow capsules, mix contents with Ora Blend SF®, Ora-Plus®, orange juice, or applesauce. Check the product leaflet detailed Instructions about how to prepare and give or take a dose of Zokinvy by mixing the contents of a Zokinvy capsule with either Ora-Blend SF, Ora-Plus, orange juice, or applesauce.
Each dose of Zokinvy mixture must be prepared fresh and taken within about 10 minutes of mixing.
Do not take Zokinvy with any juice that contains grapefruit or Seville oranges. Seville oranges may also be called bitter or sour oranges.
If you miss a dose of Zokinvy, take it as soon as possible up to 8 hours before your next scheduled dose with food. If it is less than 8 hours before your next dose of Zokinvy, skip the missed dose and take Zokinvy at your next regularly scheduled dose.
Do not take Zokinvy if you are taking:
- a strong or moderate CYP3A inhibitor or inducer
- midazolam
- lovastatin, simvastatin, and atorvastatin
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Zokinvy and other medicines may affect each other, causing serious side effects. Talk with your healthcare provider before taking any new medicines.
Before taking Zokinvy, tell your healthcare provider about all of your medical conditions, including if you:
- have kidney problems.
- have eye problems.
- are pregnant or plan to become pregnant. Zokinvy can harm your unborn baby. Women who are able to become pregnant should use effective birth control (contraception) during treatment with Zokinvy.
- are breastfeeding or plan to breastfeed. It is not known if Zokinvy can pass into your breast milk. Talk to your healthcare provider about the best way to feed your baby during treatment with Zokinvy.
Do not give Zokinvy to children under the age of 12 months because it has not been studied in this age group.
What are the possible side effects of Zokinvy?
Zokinvy may cause serious side effects, including:
Severe kidney problems. Zokinvy may cause severe kidney problems. Your healthcare provider will monitor your kidney function regularly during treatment with Zokinvy.
Eye problems. Zokinvy may cause problems with night vision. Call your healthcare provider right away if you develop any new changes in your vision.
The most common side effects of Zokinvy include:
- vomiting
- diarrhea
- decreased appetite
- nausea
- stomach-area (abdominal) pain
- tiredness
- increased liver enzyme blood test results
- weight loss
- constipation
- muscle and joint (musculoskeletal) pain
- upper respiratory tract infection
Call your healthcare provider if you continue to have nausea, vomiting, or diarrhea that leads to loss of appetite and weight loss during your treatment with Zokinvy.
Zokinvy may cause an increase in your blood pressure (hypertension). Your healthcare provider will check your blood pressure during treatment with Zokinvy. Call your healthcare provider right away if you develop any symptoms of high blood pressure including headaches, shortness of breath, nosebleeds, flushing, tiredness, dizziness, vision problems, irregular heartbeat, or chest pain.
Zokinvy may cause fertility problems in women and men, which may affect your ability to have children. Talk to your healthcare provider if you have concerns about fertility. These are not all of the possible side effects of Zokinvy.
You are encouraged to report negative side effects of prescription drugs to Eiger BioPharmaceuticals, Inc. at 1-833-267-0545 or the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
How much does Zokinvy cost?
The cost for Zokinvy oral capsule 50 mg is around $22,466 for a supply of 30 capsules, depending on the pharmacy you visit. Prices are for cash paying customers only and are not valid with insurance plans. Zokinvy is available as a brand name drug only, a generic version is not yet available.
| Quantity | Per unit | Price |
| 30 capsules of Zokinvy | $748.86 | $22,465.94 |
