Lonafarnib
Lonafarnib is a farnesyltransferase inhibitor used to treat Hutchinson-Gilford Progeria Syndrome (HGPS) and related progeroid laminopathies, rare genetic disorders characterized by accelerated aging. By preventing the farnesylation of progerin and progerin-like proteins, this medication reduces their accumulation in the nuclear membrane, which helps slow disease progression.
Lonafarnib treats Progeria and related disorders by inhibiting farnesyltransferase to prevent accumulation of the harmful progerin protein.
Key facts at a glance
- ATC
- A16AX20
- RxNorm
- 2467553
- Generic
- Lonafarnib
- Class
- atc-a16
- Brands
- Zokinvy
- Availability
- rx
- Lactation
- unknown
- Routes
- oral
- Forms
- capsule
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Treatment of Hutchinson-Gilford Progeria Syndrome (HGPS) and progeroid laminopathies with defective lamin A processing. These are rare genetic disorders causing premature aging in children.
Inhibits the enzyme farnesyltransferase, preventing the farnesylation of progerin and progerin-like proteins. This blocks their attachment to the inner nuclear membrane and reduces their toxic accumulation, which is responsible for the cellular dysfunction seen in these conditions.
Administered orally, typically twice daily with meals. Common side effects include gastrointestinal symptoms such as diarrhea, nausea, and vomiting. Requires regular monitoring for efficacy and tolerability. Treatment should be initiated and supervised by specialists familiar with progeroid disorders.
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed July 26, 2026. · Read our editorial policy