Lonafarnib
What Is Lonafarnib?[edit | edit source]
Lonafarnib (ZOKINVY) is a prescription medicine used to:
- lower the risk of death in adults and children 12 months of age or older with Hutchinson-Gilford Progeria Syndrome (HGPS), who have a certain body surface area.
- treat adults and children 12 months of age or older with certain types of Progeroid Laminopathies, who have a certain body surface area.
What are the uses of this medicine?[edit | edit source]
This medicine is indicated in patients 12 months of age and older with a body surface area (BSA) of 0.39 m2 and above:
- To reduce the risk of mortality in Hutchinson-Gilford Progeria Syndrome (HGPS)
For the treatment of processing-deficient Progeroid Laminopathies with either:
- Heterozygous LMNA mutation with progerin-like protein accumulation
- Homozygous or compound heterozygous ZMPSTE24 mutations
How does this medicine work?[edit | edit source]
- Lonafarnib inhibits farnesyl transferase to prevent farnesylation and subsequent accumulation of progerin and progerin-like proteins in the inner nuclear membrane.
Who Should Not Use this medicine ?[edit | edit source]
Do not take ZOKINVY if you are taking:
- a strong or moderate CYP3A inhibitor or inducer
- midazolam
- lovastatin or simvastatin or atorvastatin
Is this medicine FDA approved?[edit | edit source]
- It was approved for use in the United States in 2020.
How should this medicine be used?[edit | edit source]
- 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
What are the dosage forms and brand names of this medicine?[edit | edit source]
This medicine is available in fallowing doasage form:
- Available as 50 mg and 75 mg Capsules
This medicine is available in fallowing brand namesː
- ZOKINVY
What side effects can this medication cause?[edit | edit source]
Common possible side effects of this medicine include:
- vomiting
- diarrhea
- infection
- nausea
- decreased appetite
- tiredness
- upper respiratory tract infection
- stomach-area (abdominal) pain
- muscle and joint (musculoskeletal) pain
- electrolyte abnormalities
- decreased weight
- headache
- myelosuppression
- increased liver enzyme blood test results
- decreased blood bicarbonate
- cough
- hypertension
ZOKINVY may cause serious side effects, including:
- Severe kidney problems
- Eye problems
What special precautions should I follow?[edit | edit source]
- Advise females of reproductive potential of the risk to a fetus and to use effective contraception.
- Monitor for changes in electrolytes, complete blood counts, and liver enzymes.
- Monitor renal function at regular intervals
- Perform ophthalmological evaluation at regular intervals and at the onset of any new visual changes.
- Advise females and males of reproductive potential about Impaired Fertility.
What to do in case of emergency/overdose?[edit | edit source]
Can this medicine be used in pregnancy?[edit | edit source]
- There are no human data on ZOKINVY use in pregnant women to evaluate for a drug-associated risk of major birth defects, miscarriage or adverse maternal or fetal outcomes.
- Advise pregnant women of the risk to a fetus.
Can this medicine be used in children?[edit | edit source]
- The safety and effectiveness of ZOKINVY for the treatment of HGPS and processing-deficient Progeroid Laminopathies (with either heterozygous LMNA mutation with progerin-like protein accumulation or homozygous or compound heterozygous ZMPSTE24 mutations) have been established in pediatric patients 12 months of age and older.
- The safety and effectiveness of ZOKINVY in pediatric patients less than 12 months of age have not been established.
What should I know about storage and disposal of this medication?[edit | edit source]
- Store at room temperature between 68°F to 77°F (20°C to 25°C)
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