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lonafarnib

Zokinvy · ATC code 180

Key facts
Active substance
lonafarnib
ATC code
180
Authorisation
centrally authorised across the EU (EMA)
Dosage forms
Zokinvy 50 mg hard capsules / Zokinvy 75 mg hard capsules
Marketing authorisation holder
Integral Pharma Solutions EU Limited
Information as of
2026-10-28

Key facts

Lonafarnib (Zokinvy, hard capsules) is a farnesyltransferase inhibitor. It is used for Hutchinson-Gilford progeria syndrome and certain processing-deficient progeroid laminopathies.

What it is used for

  • Patients 12 months of age and older with a genetically confirmed diagnosis of Hutchinson-Gilford progeria syndrome.
  • Processing-deficient progeroid laminopathy with a heterozygous LMNA mutation with progerin-like protein accumulation, or a homozygous or compound heterozygous ZMPSTE24 mutation.

Dosing

  • Starting dose for all indications: 115 mg/m2 twice daily, body surface area by the Du Bois formula; total daily dose rounded to the nearest 25 mg and split into two doses about 12 hours apart.
  • Maintenance dose: 150 mg/m2 twice daily; timing of dose escalation in the full text.
  • Avoid grapefruit, cranberries, pomegranate and Seville (bitter) oranges.
  • Contraindicated in severe hepatic impairment (Child-Pugh C).

Contraindications

  • Hypersensitivity to the active substance, any other member of the farnesyltransferase class, or any of the excipients.
  • Concomitant use with strong CYP3A inhibitors.
  • Concomitant use of medicines predominantly metabolised by CYP3A4, such as midazolam, atorvastatin, lovastatin and simvastatin.
  • Severe hepatic impairment (Child-Pugh C).

Important warnings

  • Start as soon as the diagnosis is made; expected survival benefit is smaller when treatment starts at 10 years of age or above.
  • QTc prolongation; avoid in patients with arrhythmias, long QT syndrome or electrolyte disturbances.
  • Monitor gastrointestinal reactions and dehydration, especially in the first 4 months; correct electrolyte imbalances.
  • Measure renal function annually and perform an annual ophthalmological evaluation (nephrotoxicity and retinal toxicity in animals).
  • Avoid moderate and strong CYP3A inducers.

Side effects

  • Very common: vomiting (86%), diarrhoea (78%), increased AST (64%), increased ALT (50%), decreased appetite (41%), nausea (38%), abdominal pain, fatigue, decreased weight, constipation, upper respiratory tract infection.
  • Serious: increased ALT and AST, cerebral ischaemia (3.2%), pyrexia, dehydration.
  • Most adverse reactions occur within the first 4 weeks.

Interactions

  • Ketoconazole increased lonafarnib AUC 5.3-fold; strong CYP3A inhibitors are contraindicated.
  • Lonafarnib inhibits CYP3A4 and increases levels of CYP3A4 substrates (e.g. midazolam).
  • CYP3A inducers may reduce efficacy.

Pregnancy and breastfeeding

  • Not recommended during pregnancy.
  • Contraception: women for at least 1 week, men for at least 3 months after the final dose; add a barrier method if hormonal contraception is used.

Summary of the official product information, written with AI on 2026-10-29. Every number was checked automatically against the original text. Not reviewed by a physician – the full official text is authoritative.

Full official text →

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For patients

Package leaflet, verbatim

This product has no separate US patient information.

Authorisation and brand names by country

Medicines with exactly this active-substance composition, as listed in the national registers – alphabetical and without ranking. Whether a product is currently available is not shown here.

EU – centrally authorised (all member states)

  • ZokinvyIntegral Pharma Solutions EU Limited · EMEA/H/C/005271

Source: European Medicines Agency (EMA), product information

Brand names in 4 more European countries
France
Zokinvy
Iceland
Zokinvy
Liechtenstein
Zokinvy
Norway
Zokinvy

EMA – Article 57 product data (EU/EEA), 2026-10-29

Medelix receives no money from pharmaceutical companies. Brand names are listed alphabetically and without ranking.

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Medelix answers clinical questions from guidelines, studies and product information – every claim with its source.

Sources

  1. European Medicines Agency. Zokinvy – EPAR, Product Information. 2026-10-28. https://www.ema.europa.eu/en/documents/product-information/zokinvy-epar-product-information_en.pdf

Editorial: Medelix editorial team · last updated 2026-10-28 · How these pages are made · Report an error

Source: European Medicines Agency (EMA), product information. Not advertising, not a treatment recommendation. For questions about your own treatment, ask your doctor or pharmacist.