Lonafarnib

What Is Lonafarnib?

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?

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?

• 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 ?

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?

• It was approved for use in the United States in 2020.

How should this medicine be used?

• 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?

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?

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?

• 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.

Can this medicine be used in pregnancy?

• 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?

• 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?

• Store at room temperature between 68°F to 77°F (20°C to 25°C)

• Dailymed label info on Lonafarnib
• FDA Lonafarnib