Description
Zokinvy (lonafarnib) is an orally administered farnesyltransferase inhibitor indicated to reduce the risk of mortality in patients with Hutchinson-Gilford Progeria Syndrome (HGPS) and for the treatment of certain processing-deficient progeroid laminopathies with LMNA mutation and progerin or progerin-like protein accumulation. Zokinvy works by inhibiting the farnesylation of progerin, a mutant form of the lamin A protein, thereby reducing its toxic accumulation in the nuclear envelope and mitigating cellular damage. It is the first FDA-approved therapy specifically targeting the underlying molecular cause of HGPS.
Fact Table |
Formula | C27H31ClN4O2 |
License | US FDA (2020) |
Bioavailability | ~75% (estimated) |
Legal status | Rx-only |
Chemical Name | Lonafarnib |
Elimination half-life | ~4–6 hours |
Dosage (Strength) | 50 mg, 75 mg capsules |
Pregnancy | Not recommended; may cause fetal harm |
Brands | Zokinvy |
Protein binding | 99% |
PubChem CID | 16005130 |
MedlinePlus | a620042 |
ChEBI | 93083 |
ATC code | L04AX09 |
DrugBank | DB06435 |
KEGG | D12053 |
Routes of administration | Oral |
Directions
Zokinvy should be taken orally twice daily with food. Dosing is based on body surface area (BSA) and should be calculated by the prescribing physician. The medication should be swallowed whole with water and should not be crushed, chewed, or dissolved. If vomiting occurs within 30 minutes of a dose, the dose may be repeated. Close monitoring is necessary to adjust dosing and manage adverse reactions. A gradual dose ramp-up is recommended to minimize gastrointestinal side effects.
Ingredients
Active Ingredient: Lonafarnib Inactive Ingredients: Microcrystalline cellulose, mannitol, sodium starch glycolate, magnesium stearate, and a film-coating containing polyvinyl alcohol, titanium dioxide, polyethylene glycol, talc, and iron oxide yellow.
Contraindications
Zokinvy is contraindicated in patients taking strong or moderate CYP3A inhibitors or inducers, or drugs that are OATP1B1 or OATP1B3 substrates due to the risk of serious drug interactions. It is also contraindicated in patients with known hypersensitivity to lonafarnib or any component of the formulation.
Cautions
Hepatic toxicity, gastrointestinal effects (including nausea, vomiting, and diarrhea), and electrolyte abnormalities may occur and require monitoring and management. Liver function tests, serum electrolytes, and renal function should be monitored regularly. Avoid use during pregnancy due to potential teratogenic effects. Effective contraception is advised during treatment and for a period following discontinuation. Use caution in patients with hepatic or renal impairment.
Side Effects
Common side effects may include:
- Electrolyte imbalances (e.g., hypokalemia, hypophosphatemia)
Serious adverse events may include QT prolongation, hepatotoxicity, and significant gastrointestinal toxicity requiring dose adjustments or discontinuation.
IMPORTANT NOTE:
The above information is intended to increase awareness of health information and does not suggest treatment or diagnosis. This information is not a substitute for individual medical attention and should not be construed to indicate that use of the drug is safe, appropriate, or effective for you. See your health care professional for medical advice and treatment.
Product Code : 14193