Zokinvy (lonafarnib)
An approved treatment for Progeria.
Drug facts
The same compound appears under different names depending on the context. Here is how to identify Lonafarnib wherever you encounter it, plus the key facts at a glance.
- Generic name
- Lonafarnib
- Brand name
- Zokinvy
- Drug class
- Farnesyltransferase inhibitor
- Manufacturer
- Sentynl Therapeutics (a Zydus Lifesciences company)
- How it's taken
- Oral capsules twice a day with the morning and evening meals
A twice-daily capsule and the first FDA-approved treatment for Hutchinson-Gilford progeria syndrome (HGPS), approved on November 20, 2020 to reduce the risk of death[1][3].
- Zokinvy is also approved for certain processing-deficient progeroid laminopathies, related conditions caused by particular LMNA or ZMPSTE24 gene changes.
- The label covers people 12 months and older with a body surface area, a measure of body size worked out from height and weight, of at least 0.39 square meters[1].
- Zokinvy has no boxed warning.
- Sentynl Therapeutics, a Zydus Lifesciences company, has held the worldwide rights since buying Zokinvy from Eiger BioPharmaceuticals in May 2024[5].
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Where Lonafarnib fits
Lonafarnib, sold as Zokinvy, is the first FDA-approved medicine for Hutchinson-Gilford progeria syndrome and is approved to reduce the risk of death in HGPS and to treat certain processing-deficient progeroid laminopathies[1][3].
- The Progeria Research Foundation calls lonafarnib the current standard of care for the children in its trials, and it publishes a Progeria Clinical Care Handbook for families and doctors[9].
- No other medicine is FDA approved for progeria.
- Progerinin, from PRG Science & Technology, was tested added to lonafarnib in a 10-person Phase 2 study (NCT06775041) that ClinicalTrials.gov lists as completed, with no results posted[8][10].
- Zokinvy's approval rests on a comparison with untreated children from a natural history group, and no trial has compared lonafarnib head to head with another treatment[1].
- A question to bring to the care team is how often ECGs, eye exams, kidney tests and blood tests will be scheduled, and which of a child's other medicines interact with lonafarnib.
How Lonafarnib works
In classic HGPS, a change in the LMNA gene makes cells produce progerin, a faulty form of lamin A, the protein that gives the cell's nucleus its shape. Progerin keeps a fatty tag called a farnesyl group, and the label explains that this tag lets progerin and progerin-like proteins collect in the inner membrane of the nucleus[1]. Lonafarnib blocks farnesyltransferase, the enzyme that attaches the tag, which prevents that buildup[1].
The same tag problem drives processing-deficient progeroid laminopathies, which is why the label covers them. The label says Zokinvy is not indicated for other progeroid syndromes or for processing-proficient progeroid laminopathies, because its mechanism would not be expected to help there[1].
Side effects and safety
- QTc prolongation (heart rhythm). Zokinvy lengthens the QTc interval, which raises the risk of torsade de pointes, other serious arrhythmias and sudden death. An ECG is done before starting and during treatment, blood salts are checked and corrected, and Zokinvy is held when the QTc reaches 500msec until it falls below 470msec.
- Drug interactions. Strong CYP3A inhibitors, strong or moderate CYP3A inducers, midazolam, and lovastatin, simvastatin or atorvastatin cannot be combined with Zokinvy. Other medicines that lengthen the QTc, sensitive CYP3A substrates and CYP2C19 substrates are avoided, and loperamide starts at no more than 1mg once a day.
- Lab changes. Blood salt changes (43%), lower blood counts (35%) and raised AST (35%) or ALT (27%) in the trials. Electrolytes, blood counts and liver enzymes are checked periodically.
- Kidney toxicity in rats. Lonafarnib harmed the kidneys of rats at exposures close to the human dose, and the label asks for kidney function tests at regular intervals.
- Retinal toxicity in monkeys. Lonafarnib reduced rod-dependent, low-light vision in monkeys. Eye exams are done at regular intervals and whenever vision changes, and the label tells families to watch for trouble seeing at night.
- Fertility. Lonafarnib reduced fertility in female and male rats and harmed the male reproductive tract in monkeys. The effects on puberty and fertility in people have not been adequately studied.
- Pregnancy. Animal studies showed harm to the fetus, and there are no human data. The label advises effective birth control for people who could become pregnant.
- ECG before starting, during treatment and when another QTc-lengthening medicine is added
- Blood salts (electrolytes) before starting and during treatment, corrected when abnormal
- Blood counts and liver enzymes periodically
- Kidney function at regular intervals
- Eye exams at regular intervals and with any new vision change
- Weight and fluids when vomiting or diarrhea lead to dehydration or weight loss
- Blood pressure during treatment
Zokinvy has no boxed warning[1]. Stomach and gut side effects were the most common in the 2 trials behind the label, which treated 63 children and young people aged 2 to 17 for a median of 2.2 years: vomiting in 90%, diarrhea in 81% and nausea in 56%[1]. Most cases were mild or moderate. In the first trial, diarrhea affected 82% of children in the first 4 months of treatment and 11% by the end, and 4 patients stopped Zokinvy, mostly because of nausea or vomiting[1].
Other common reactions were infections (78%), upper respiratory infections (51%), fatigue (51%), less appetite (53%), belly pain (48%), muscle and bone pain (48%), headache (37%) and weight loss (37%)[1]. Lab changes were also common: blood salt (electrolyte) changes in 43%, lower blood cell counts (myelosuppression) in 35%, raised liver enzymes AST in 35% and ALT in 27%, and the label says these often improved while treatment continued[1].
Low blood bicarbonate was reported in 33%. Cough affected 33% and high blood pressure 29%, and the label reports cerebral ischemia, reduced blood flow in the brain, in 11%[1]. Zokinvy lengthens the QTc interval on an ECG, a heart rhythm measure, which raises the risk of a dangerous rhythm called torsade de pointes, and it interacts with many medicines because it strongly blocks the liver enzyme CYP3A[1].
In animal studies, lonafarnib harmed the kidneys of rats, reduced low-light (night) vision in monkeys, reduced fertility in rats and harmed the male reproductive tract in monkeys, and the label asks for regular kidney tests and eye exams[1]. Based on animal studies, Zokinvy can harm an unborn baby, and the label advises effective birth control for people who could become pregnant[1].
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Lonafarnib
Zokinvy is a capsule taken by mouth twice a day with the morning and evening meals, and the dose is set by body surface area[1]. Treatment starts at 115mg/m2 twice a day and rises to 150mg/m2 twice a day after 4 months, with each total daily dose rounded to the nearest 25mg[1]. Capsules come in 50mg (yellow) and 75mg (light orange), in bottles of 30, and the label's tables give the number of each capsule for every body size from 0.39 to 1 square meter[1].
Capsules are swallowed whole with water. For a child who cannot swallow them, the contents can be mixed with Ora Blend SF or Ora-Plus, or with orange juice or applesauce, made fresh and taken within about 10 minutes; juice with grapefruit or Seville oranges is not used[1]. A missed dose is taken with food if the next dose is more than 8 hours away, and skipped otherwise[1].
When repeated vomiting or diarrhea causes dehydration or weight loss at the higher dose, the dose can go back to 115mg/m2 twice a day. Zokinvy is paused for 10 to 14 days before and 2 days after any use of midazolam, a sedative[1].
Availability and cost
Only available as the brand-name product.
An orphan drug taken twice a day for life, dosed by body size. Neither the label nor zokinvy.com gives a price. Drugs@FDA lists only Sentynl's application, with no approved generic.
Help paying for Zokinvy
The maker of Zokinvy publishes no copay card, but its support program can check your insurance and explain any help that exists. Drugmaker copay cards can't be used with Medicare, Medicaid or TRICARE, but charity funds often can. Of the 2 charity funds for Progeria, 2 were open on October 26, 2026. Pick your insurance below to see which offers fit you.
- Insurance and case manager help
The Sentynl Cares team checks insurance coverage, helps with financial support options and appeals, and connects families to the pharmacy that dispenses Zokinvy. The page lists nurses, pharmacists, reimbursement and appeals specialists, alternate funding and co-pay assistance.
The official page does not say who qualifies. Ask the program. · source
Good to know: Call Monday to Friday, 8 AM to 8 PM ET. US residents only. The page names co-pay assistance but publishes no terms, maximums, income limits or free-drug rules, and does not name the pharmacy. Enrollment uses the Sentynl Cares | Zokinvy Enrollment Form.
Charity funds for Progeria
- From a charity · NORD RareCareProgeria & Progeroid Laminopathies Copay Assistance fundOpen
Pays for: Insurance premiums and copays.
The foundation says: “Accepting new applications and re-enrollments for current year” - From a charity · NORD RareCareProgeria & Progeroid Laminopathies Medical Assistance fundOpen
Pays for: Medical and medication costs.
The foundation says: “Accepting new applications and re-enrollments for current year”
Clinical trial results
No randomized trial tested Zokinvy. The FDA approval rests on an observational survival study that compared children treated in 2 single-arm Phase 2 trials with matched untreated children from a separate natural history group, matched by gene type, sex and continent[1]. Study 1 (NCT00425607) treated 28 children, starting at 115mg/m2 twice a day and rising to 150mg/m2, for 24 to 30 months[1][6].
Study 2 (NCT00916747) first gave lonafarnib with zoledronic acid and pravastatin, then enrolled 35 children who had never been treated to take lonafarnib alone at 150mg/m2 twice a day for up to 3 years[1][7]. Across 62 treated children and 62 matched untreated children, deaths through 3 years of follow-up were 5 (8.1%) against 12 (19.4%), and through the last follow-up of up to 11 years 21 (33.9%) against 25 (40.3%)[1].
The label reports that average survival was about 3 months longer through the first 3 years and 2.5 years longer through the last follow-up (8.0 against 5.5 years), with hazard ratios of 0.30 and 0.40[1]. The label's confidence interval for the 3-year difference in average survival, -0.03 to 0.50 years, includes zero, while the interval for the last follow-up, 0.8 to 4.1 years, does not[1].
The Progeria Research Foundation reports that a later trial of lonafarnib with everolimus enrolled 60 children from 27 countries between 2017 and 2022 and that its data are still being analyzed[9].
Development history
Eiger BioPharmaceuticals submitted the new drug application on March 20, 2020, and the FDA approved Zokinvy on November 20, 2020 under NDA 213969, as a new molecular entity with priority review and orphan status[4][3]. The Progeria Research Foundation describes its first lonafarnib trial, with drug supplied by Merck & Co., as the first known effective treatment for children with progeria[11].
Eiger filed for Chapter 11 bankruptcy on April 1, 2024, and Sentynl Therapeutics, wholly owned by Zydus Lifesciences, bought the worldwide rights to Zokinvy in a sale that closed on May 3, 2024, according to Sentynl's release[5]. The FDA approved label updates in March 2024 that changed the dosing section on side effects and drug interactions and the heart rhythm warning, and another label update on November 12, 2026[3][2]. The label revised in November 2026 reports the current safety and dosing information[1].
Explore Progeria trials
Common questions about Lonafarnib
▸What is Lonafarnib (Zokinvy)?
A twice-daily capsule and the first FDA-approved treatment for Hutchinson-Gilford progeria syndrome (HGPS), approved on November 20, 2020 to reduce the risk of death[1][3]. Zokinvy is also approved for certain processing-deficient progeroid laminopathies, related conditions caused by particular LMNA or ZMPSTE24 gene changes. The label covers people 12 months and older with a body surface area, a measure of body size worked out from height and weight, of at least 0.39 square meters[1]. Zokinvy has no boxed warning. Sentynl Therapeutics, a Zydus Lifesciences company, has held the worldwide rights since buying Zokinvy from Eiger BioPharmaceuticals in May 2024[5].
▸How does Lonafarnib work?
In classic HGPS, a change in the LMNA gene makes cells produce progerin, a faulty form of lamin A, the protein that gives the cell's nucleus its shape. Progerin keeps a fatty tag called a farnesyl group, and the label explains that this tag lets progerin and progerin-like proteins collect in the inner membrane of the nucleus[1]. Lonafarnib blocks farnesyltransferase, the enzyme that attaches the tag, which prevents that buildup[1]. The same tag problem drives processing-deficient progeroid laminopathies, which is why the label covers them. The label says Zokinvy is not indicated for other progeroid syndromes or for processing-proficient progeroid laminopathies, because its mechanism would not be expected to help there[1].
▸What are the side effects of Lonafarnib?
Zokinvy has no boxed warning[1]. Stomach and gut side effects were the most common in the 2 trials behind the label, which treated 63 children and young people aged 2 to 17 for a median of 2.2 years: vomiting in 90%, diarrhea in 81% and nausea in 56%[1]. Most cases were mild or moderate. In the first trial, diarrhea affected 82% of children in the first 4 months of treatment and 11% by the end, and 4 patients stopped Zokinvy, mostly because of nausea or vomiting[1]. Other common reactions were infections (78%), upper respiratory infections (51%), fatigue (51%), less appetite (53%), belly pain (48%), muscle and bone pain (48%), headache (37%) and weight loss (37%)[1]. Lab changes were also common: blood salt (electrolyte) changes in 43%, lower blood cell counts (myelosuppression) in 35%, raised liver enzymes AST in 35% and ALT in 27%, and the label says these often improved while treatment continued[1]. Low blood bicarbonate was reported in 33%. Cough affected 33% and high blood pressure 29%, and the label reports cerebral ischemia, reduced blood flow in the brain, in 11%[1]. Zokinvy lengthens the QTc interval on an ECG, a heart rhythm measure, which raises the risk of a dangerous rhythm called torsade de pointes, and it interacts with many medicines because it strongly blocks the liver enzyme CYP3A[1]. In animal studies, lonafarnib harmed the kidneys of rats, reduced low-light (night) vision in monkeys, reduced fertility in rats and harmed the male reproductive tract in monkeys, and the label asks for regular kidney tests and eye exams[1]. Based on animal studies, Zokinvy can harm an unborn baby, and the label advises effective birth control for people who could become pregnant[1].
▸How is Lonafarnib taken?
Zokinvy is a capsule taken by mouth twice a day with the morning and evening meals, and the dose is set by body surface area[1]. Treatment starts at 115mg/m2 twice a day and rises to 150mg/m2 twice a day after 4 months, with each total daily dose rounded to the nearest 25mg[1]. Capsules come in 50mg (yellow) and 75mg (light orange), in bottles of 30, and the label's tables give the number of each capsule for every body size from 0.39 to 1 square meter[1]. Capsules are swallowed whole with water. For a child who cannot swallow them, the contents can be mixed with Ora Blend SF or Ora-Plus, or with orange juice or applesauce, made fresh and taken within about 10 minutes; juice with grapefruit or Seville oranges is not used[1]. A missed dose is taken with food if the next dose is more than 8 hours away, and skipped otherwise[1]. When repeated vomiting or diarrhea causes dehydration or weight loss at the higher dose, the dose can go back to 115mg/m2 twice a day. Zokinvy is paused for 10 to 14 days before and 2 days after any use of midazolam, a sedative[1].
▸Is Lonafarnib FDA approved?
Yes. The FDA approved Zokinvy on November 20, 2020 to reduce the risk of death in Hutchinson-Gilford progeria syndrome[1].
Who: people 12 months and older with a body surface area of at least 0.39 square meters.
Also approved for: processing-deficient progeroid laminopathies with certain LMNA or ZMPSTE24 variants.
Not approved for: other progeroid syndromes or processing-proficient progeroid laminopathies.
Source: Zokinvy prescribing information, November 2026; FDA approval letter, November 20, 2020.
▸How much does Zokinvy extend life in progeria?
In the comparison behind the FDA approval, children treated with Zokinvy lived about 2.5 years longer on average over up to 11 years of follow-up than matched untreated children[1].
First 3 years: average survival was about 3 months longer, 2.8 against 2.6 years, and deaths were 5 of 62 against 12 of 62.
Last follow-up: average survival was 8.0 against 5.5 years, and deaths were 21 of 62 against 25 of 62.
How it was measured: children from 2 single-arm Phase 2 trials were matched with untreated children from a separate natural history group; there was no placebo group.
Who was studied: 62 children with HGPS aged 2 to 17 when treatment began.
Source: Zokinvy prescribing information, November 2026.
▸What medicines can't be taken with Zokinvy?
The label rules out strong CYP3A inhibitors, strong or moderate CYP3A inducers, midazolam, and the statins lovastatin, simvastatin and atorvastatin[1].
Why: lonafarnib is broken down by CYP3A and also strongly blocks it, which can raise its own levels or other drugs' levels, or lower its effect.
Midazolam: Zokinvy is paused for 10 to 14 days before and 2 days after a dose.
Avoided: other medicines that lengthen the QTc on an ECG, sensitive CYP3A substrates, CYP2C19 substrates, and grapefruit or Seville oranges.
Loperamide: no more than 1mg once a day when first combined, raised slowly.
Monitored: P-gp substrates such as digoxin and dabigatran.
Ask: a pharmacist or doctor can check each of a child's medicines against the label.
Source: Zokinvy prescribing information, November 2026.
▸Can Zokinvy capsules be opened for a child who can't swallow them?
Yes. The label allows the capsule contents to be mixed with a liquid or applesauce[1].
Preferred mixers: Ora Blend SF or Ora-Plus, 5mL to 10mL per capsule.
Other options: orange juice, 5mL to 10mL per capsule, or 1 to 2 teaspoons of applesauce per capsule, for children who cannot get or tolerate the preferred mixers.
Not used: juice containing grapefruit or Seville oranges.
Timing: mixed fresh for each dose and taken within about 10 minutes, with the morning and evening meals.
Source: Zokinvy prescribing information, November 2026.
Sources and references
Every factual claim on this page is drawn from the public sources listed below. Click any reference to open the original document.
- Sentynl Therapeutics via FDA (accessdata.fda.gov) · 2026-11-12. ZOKINVY (lonafarnib) capsules, for oral use: prescribing information. https://www.accessdata.fda.gov/drugsatfda_docs/label/2026/213969s004lbl.pdf
- Sentynl Therapeutics via DailyMed · 2025-07-02. ZOKINVY- lonafarnib capsule. https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=97391d0c-943e-4c5a-8423-68e163aa7f50
- FDA · 2020-11-20. ZOKINVY approval history, NDA 213969. https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=213969
- FDA · 2020-11-20. Zokinvy (lonafarnib) Approval Letter. https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2020/213969Orig1s000ltr.pdf
- Sentynl Therapeutics via PR Newswire (BioSpace) · 2024-05-03. Sentynl Therapeutics Announces Global Acquisition of Zokinvy® (Lonafarnib) for Treatment of Hutchinson-Gilford Progeria Syndrome from Eiger BioPharmaceuticals. https://www.biospace.com/sentynl-therapeutics-announces-global-acquisition-of-zokinvy-lonafarnib-for-treatment-of-hutchinson-gilford-progeria-syndrome-from-eiger-biopharmaceuticals
- ClinicalTrials.gov. Phase II Trial of Lonafarnib (a Farnesyltransferase Inhibitor) for Progeria. https://clinicaltrials.gov/study/NCT00425607
- ClinicalTrials.gov. Study of Zoledronic Acid, Pravastatin, and Lonafarnib for Patients With Progeria. https://clinicaltrials.gov/study/NCT00916747
- ClinicalTrials.gov. Study to Determine Optimal Dose and Evaluate Safety, Tolerability, and Pharmacokinetics of Progerinin in Patients With Hutchinson-Gilford Progeria Syndrome (HGPS). https://clinicaltrials.gov/study/NCT06775041
- The Progeria Research Foundation. Clinical trials | The Progeria Research Foundation. https://www.progeriaresearch.org/clinical-trials/
- The Progeria Research Foundation. Progerinin trial | The Progeria Research Foundation. https://www.progeriaresearch.org/progerinin_trial/
- The Progeria Research Foundation. treatment | The Progeria Research Foundation. https://www.progeriaresearch.org/first-ever-progeria-treatment/
- Sentynl Therapeutics. Zokinvy Personalized Care from Sentynl Cares | Zokinvy (lonafarnib) Patient. https://www.zokinvy.com/sentynl-cares