Zokinvy
Zokinvy (lonafarnib) is an oral medication classified as a farnesyltransferase inhibitor, designed to treat Hutchinson-Gilford Progeria Syndrome (HGPS) and certain processing-deficient progeroid laminopathies—rare genetic disorders characterized by accelerated aging. It works by reducing the accumulation of a defective protein called progerin that causes the rapid aging and health complications seen in these conditions. This is the first FDA-approved treatment specifically for these rare progeroid syndromes.
Zokinvy is an oral farnesyltransferase inhibitor approved to treat rare genetic aging disorders like Hutchinson-Gilford Progeria Syndrome by reducing progerin protein accumulation.
Key facts at a glance
- RxNorm
- 2467583
- Generic
- Zokinvy
- Class
- brand-name
- Brands
- —
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- —
- 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.
Zokinvy is indicated for the treatment of Hutchinson-Gilford Progeria Syndrome (HGPS) and for patients 12 months and older with certain processing-deficient progeroid laminopathies with heterozygous LMNA mutation with progerin-like protein accumulation, or homozygous or compound heterozygous ZMPSTE24 mutations.
Zokinvy is a farnesyltransferase inhibitor that works by blocking the enzyme responsible for attaching farnesyl groups to proteins. In progeria and related disorders, a defective protein called progerin accumulates and causes cellular damage. By inhibiting this process, Zokinvy helps reduce progerin accumulation and may slow the progression of disease symptoms.
Zokinvy is available only through a restricted distribution program due to its specific indication for rare diseases. Common side effects may include gastrointestinal symptoms such as vomiting, diarrhea, and nausea. Patients should be monitored for potential liver enzyme elevations and other adverse effects. Treatment should be initiated and supervised by physicians experienced in managing patients with progeroid syndromes.
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 August 27, 2026. · Read our editorial policy