Human medicines European public assessment report (EPAR): Zokinvy, lonafarnib, Date of authorisation: 18/07/2022, Revision: 4, Status: Authorised

Zokinvy RSS Authorised This medicine is authorised for use in the European Union lonafarnib Medicine Human Authorised Application under evaluation CHMP opinion European Commission decision Overview Zokinvy is a medicine used to treat patients of 12 months and older who are affected by the following rare diseases in which features resembling aging appear in childhood: Hutchinson-Gilford progeria syndrome; processing-deficient progeroid laminopathies. The diseases that Zokinvy is used to treat are rare and Zokinvy was designated as ‘orphan medicine’ on 14 December 2018 for Hutchinson-Gilford progeria syndrome. Zokinvy contains the active substance lonafarnib. Expand section Collapse section How is Zokinvy used? Zokinvy can only be obtained with a prescription and treatment should be initiated by doctors with experience in the treatment of patients with premature aging or rare metabolic conditions. Zokinvy is available as capsules to be taken with food twice a day. The starting daily dose (which ranges from 75 to 225 mg) depends on the height and weight of the patient. After 4 months of treatment, the patient may start taking a higher (maintenance) dose. For more information about using Zokinvy, see the package leaflet or contact your doctor or pharmacist. How does Zokinvy work? Patients with Hutchinson-Gilford progeria syndrome and with processing-deficient progeroid laminopathies have an accumulation of abnormal forms of progerin or progerin-like proteins, which causes damage to cells and leads to symptoms of aging early in life. Zokinvy prevents a chemical reaction involved in the formation of these abnormal proteins, thereby helping to improve symptoms of the diseases. What benefits of Zokinvy have been shown in studies? Two main studies have shown that Zokinvy prolongs the life of patients with Hutchinson-Gilford progeria syndrome and with processing-deficient progeroid laminopathies. The studies involved 62 patients who were given Zokinvy. Three years after start of treatment with Zokinvy only, the patients lived between 2.5 months and about half a year longer than the 62 patients who did not participate in the studies and were not given Zokinvy. At the time of the last follow-up (about 11 years after starting treatment), patients given Zokinvy (and possibly additional treatments) lived an average of 4.3 years longer than untreated patients. However, given the limited data available, the extra years lived could be as low as 2.6 years. What are the risks associated with Zokinvy? The most common side effects with Zokinvy (which may affect more than 1 in 10 people) are vomiting, diarrhoea, increased levels of liver enzymes, decreased appetite, nausea, abdominal pain, tiredness, weight loss, constipation and upper respiratory tract infection (nose and throat infection). The most common serious side effects with Zokinvy (which may affect up to 1 in 10 people) are increased levels of liver enzymes, cerebral ischaemia (reduced blood supply to the brain), fever and dehydration. Why is Zokinvy authorised in the EU? At the time of the authorisation of Zokinvy there were no other medicines for treating Hutchinson-Gilford progeria syndrome and processing-deficient progeroid laminopathies. The results from the studies of Zokinvy showed that this medicine can prolong the life of patients with these conditions. The most common side effects, such as diarrhoea, nausea, and vomiting, occurred mainly in the first 4 months of treatment and were manageable. The European Medicines Agency therefore decided that Zokinvy’s benefits are greater than its risks and it can be authorised for use in the EU under ‘exceptional circumstances’. This is because it has not been possible to obtain complete information about Zokinvy due to the rarity of the disease. Every year, the European Medicines Agency will review any new information that becomes available, and this overview will be updated as necessary. What information is still awaited for Zokinvy? Since Zokinvy has been authorised under exceptional circumstances, the company that markets Zokinvy will provide data from a registry of patients treated with the medicine to further evaluate the safety and effectiveness of Zokinvy as well as the quality of life of patients. What measures are being taken to ensure the safe and effective use of Zokinvy? Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Zokinvy have been included in the summary of product characteristics and the package leaflet. As for all medicines, data on the use of Zokinvy are continuously monitored. Suspected side effects reported with Zokinvy are carefully evaluated and any necessary action taken to protect patients. Other information about Zokinvy Zokinvy received a marketing authorisation valid throughout the EU on 18 July 2022. Zokinvy: EPAR - Medicine overview Reference Number: EMA/351469/2022 English (EN) (143.11 KB - PDF) First published: 01/08/2022 View Other languages (22) български (BG) (169.05 KB - PDF) First published: 01/08/2022 View español (ES) (142.52 KB - PDF) First published: 01/08/2022 View čeština (CS) (155.79 KB - PDF) First published: 01/08/2022 View dansk (DA) (142.26 KB - PDF) First published: 01/08/2022 View Deutsch (DE) (146 KB - PDF) First published: 01/08/2022 View eesti keel (ET) (130.56 KB - PDF) First published: 01/08/2022 View ελληνικά (EL) (167.52 KB - PDF) First published: 01/08/2022 View français (FR) (143.64 KB - PDF) First published: 01/08/2022 View hrvatski (HR) (164.14 KB - PDF) First published: 01/08/2022 View italiano (IT) (141.72 KB - PDF) First published: 01/08/2022 View latviešu valoda (LV) (171.3 KB - PDF) First published: 01/08/2022 View lietuvių kalba (LT) (166.04 KB - PDF) First published: 01/08/2022 View magyar (HU) (164.14 KB - PDF) First published: 01/08/2022 View Malti (MT) (168.95 KB - PDF) First published: 01/08/2022 View Nederlands (NL) (144.74 KB - PDF) First published: 01/08/2022 View polski (PL) (171.16 KB - PDF) First published: 01/08/2022 View português (PT) (132.88 KB - PDF) First published: 01/08/2022 View română (RO) (162.48 KB - PDF) First published: 01/08/2022 View slovenčina (SK) (155.81 KB - PDF) First published: 01/08/2022 View slovenščina (SL) (163.67 KB - PDF) First published: 01/08/2022 View Suomi (FI) (139.97 KB - PDF) First published: 01/08/2022 View svenska (SV) (140.96 KB - PDF) First published: 01/08/2022 View Zokinvy : EPAR - Risk-management-plan summary English (EN) (163.88 KB - PDF) First published: 01/08/2022 View Product information Zokinvy : EPAR - Product information English (EN) (1.18 MB - PDF) First published: 01/08/2022 Last updated: 07/04/2026 View Other languages (24) български (BG) (1.51 MB - PDF) First published: 01/08/2022 Last updated: 07/04/2026 View español (ES) (1.32 MB - PDF) First published: 01/08/2022 Last updated: 07/04/2026 View čeština (CS) (1.25 MB - PDF) First published: 01/08/2022 Last updated: 07/04/2026 View dansk (DA) (1.17 MB - PDF) First published: 01/08/2022 Last updated: 07/04/2026 View Deutsch (DE) (1.29 MB - PDF) First published: 01/08/2022 Last updated: 07/04/2026 View eesti keel (ET) (980.39 KB - PDF) First published: 01/08/2022 Last updated: 07/04/2026 View ελληνικά (EL) (1.31 MB - PDF) First published: 01/08/2022 Last updated: 07/04/2026 View français (FR) (1.02 MB - PDF) First published: 01/08/2022 Last updated: 07/04/2026 View hrvatski (HR) (938.86 KB - PDF) First published: 01/08/2022 Last updated: 07/04/2026 View íslenska (IS) (1.1 MB - PDF) First published: 01/08/2022 Last updated: 07/04/2026 View italiano (IT) (1.24 MB - PDF) First published: 01/08/2022 Last updated: 07/04/2026 View latviešu valoda (LV) (1.24 MB - PDF) First published: 01/08/2022 Last updated: 07/04/2026 View lietuvių kalba (LT) (1.25 MB - PDF) First published: 01/08/2022 Last updated: 07/04/2026 View magyar (HU) (1.33 MB - PDF) First published: 01/08/2022 Last updated: 07/04/2026 View Malti (MT) (996.56 KB - PDF) First published: 01/08/2022 Last updated: 07/04/2026 View Nederlands (NL) (1.23 MB - PDF) First published: 01/08/2022 Last updated: 07/04/2026 View norsk (NO) (1.05 MB - PDF) First published: 01/08/2022 Last updated: 07/04/2026 View polski (PL) (1.28 MB - PDF) First published: 01/08/2022 Last updated: 07/04/2026 View português (PT) (1.21 MB - PDF) First published: 01/08/2022 Last updated: 07/04/2026 View română (RO) (1.27 MB - PDF) First published: 01/08/2022 Last updated: 07/04/2026 View slovenčina (SK) (1.14 MB - PDF) First published: 01/08/2022 Last updated: 07/04/2026 View slovenščina (SL) (1.29 MB - PDF) First published: 01/08/2022 Last updated: 07/04/2026 View Suomi (FI) (1.19 MB - PDF) First published: 01/08/2022 Last updated: 07/04/2026 View svenska (SV) (939.34 KB - PDF) First published: 01/08/2022 Last updated: 07/04/2026 View Latest procedure affecting product information: VR/0000269652 20/11/2025 This medicine’s product information is available in all official EU languages . Select 'available languages' to access the language you need. Product information documents contain: summary of product characteristics (annex I); manufacturing authorisation holder responsible for batch release (annex IIA); conditions of the marketing authorisation (annex IIB); labelling (annex IIIA); package leaflet (annex IIIB). Zokinvy : EPAR - All authorised presentations English (EN) (50.1 KB - PDF) First published: 01/08/2022 View Other languages (24) български (BG) (61.34 KB - PDF) First published: 01/08/2022 View español (ES) (55.08 KB - PDF) First published: 01/08/2022 View čeština (CS) (61.25 KB - PDF) First published: 01/08/2022 View dansk (DA) (67.85 KB - PDF) First published: 01/08/2022 View Deutsch (DE) (55.71 KB - PDF) First published: 01/08/2022 View eesti keel (ET) (54.83 KB - PDF) First published: 01/08/2022 View ελληνικά (EL) (70.41 KB - PDF) First published: 01/08/2022 View français (FR) (54.68 KB - PDF) First published: 01/08/2022 View hrvatski (HR) (70.13 KB - PDF) First published: 01/08/2022 View íslenska (IS) (50.89 KB - PDF) First published: 01/08/2022 View italiano (IT) (54.73 KB - PDF) First published: 01/08/2022 View latviešu valoda (LV) (68.02 KB - PDF) First published: 01/08/2022 View lietuvių kalba (LT) (59.35 KB - PDF) First published: 01/08/2022 View magyar (HU) (57.59 KB - PDF) First published: 01/08/2022 View Malti (MT) (70.44 KB - PDF) First published: 01/08/2022 View Nederlands (NL) (53.56 KB - PDF) First published: 01/08/2022 View norsk (NO) (49.81 KB - PDF) First published: 01/08/2022 View polski (PL) (70.7 KB - PDF) First published: 01/08/2022 View português (PT) (53.18 KB - PDF) First published: 01/08/2022 View română (RO) (59.39 KB - PDF) First published: 01/08/2022 View slovenčina (SK) (69.61 KB - PDF) First published: 01/08/2022 View slovenščina (SL) (61.14 KB - PDF) First published: 01/08/2022 View Suomi (FI) (54.01 KB - PDF) First published: 01/08/2022 View svenska (SV) (52.99 KB - PDF) First published: 01/08/2022 View Product details Name of medicine Zokinvy Active substance Lonafarnib International non-proprietary name (INN) or common name lonafarnib Therapeutic area (MeSH) Progeria Laminopathies Pharmacotherapeutic group Other alimentary tract and metabolism products Therapeutic indication Zokinvy is indicated for the treatment of patients 12 months of age and older with a genetically confirmed diagnosis of Hutchinson-Gilford progeria syndrome or a processing-deficient progeroid laminopathy associated with either a heterozygous LMNA mutation with progerin-like protein accumulation or a homozygous or compound heterozygous ZMPSTE24 mutation. Authorisation details EMA product number EMEA/H/C/005271 Additional monitoring This medicine is under additional monitoring, meaning that it is monitored even more intensively than other medicines. For more information, see Medicines under additional monitoring . Exceptional circumstances This medicine was authorised under exceptional circumstances, because the applicant was unable to provide comprehensive data on the efficacy and safety of the medicine under normal conditions of use. This can happen because the condition to be treated is rare or because collection of full information is not possible or is unethical. For more information, see Pre-authorisation guidance . Orphan This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation . Marketing authorisation holder TMC Pharma (EU) Limited G24A Arc Labs Research and Innovation Centre SETU West Campus, Carriganore Waterford X91 P20H IRELAND Opinion adopted 19/05/2022 Marketing authorisation issued 18/07/2022 Revision 4 Assessment history Expand section Collapse section Changes since initial authorisation of medicine Zokinvy : EPAR - Procedural steps taken and scientific information after authorisation English (EN) (151.97 KB - PDF) First published: 07/04/2026 View Zokinvy : EPAR - Procedural steps taken and scientific information after authorisation (archive) English (EN) (150.85 KB - PDF) First published: 24/03/2023 Last updated: 07/04/2026 View Initial marketing authorisation documents Zokinvy : Orphan maintenance assessment report (initial authorisation) Adopted English (EN) (214.95 KB - PDF) First published: 01/08/2022 View Zokinvy : EPAR - Public assessment report Adopted Reference Number: EMA/656670/2022 Rev.1 English (EN) (1.85 MB - PDF) First published: 01/08/2022 Last updated: 13/12/2022 View CHMP summary of positive opinion for Zokinvy Adopted Reference Number: EMA/CHMP/264223/2022 English (EN) (160.93 KB - PDF) First published: 20/05/2022 View News on Zokinvy Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 16-19 May 2022 20/05/2022 First treatment for children with Progeria or progeroid like syndromes (rare premature aging syndromes) 20/05/2022 More information on Zokinvy EU/3/18/2118 - orphan designation for treatment of Hutchinson-Gilford progeria This page was last updated on 07/04/2026 Share this page