Lonafarnib

From WikiMD's Food, Medicine & Wellness Encyclopedia

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:

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)
Lonafarnib Resources
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