Givlaari
Authorised
This medicine is authorised for use in the European Union
givosiran
MedicineHumanAuthorised
Overview
Givlaari is a medicine for treating acute hepatic porphyria in patients aged 12 years or over.
Acute hepatic porphyria is a rare genetic condition in which the liver cannot properly produce a substance called haem. As a result, substances used to make haem build up in the body, causing attacks of severe abdominal pain, vomiting and nervous system disorders.
Acute hepatic porphyria is rare, and Givlaari was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 29 August 2016.
Givlaari is given by injection under the skin once a month. The dose depends on the patient’s weight.
The medicine can only be obtained with a prescription and treatment should be started by a healthcare professional experienced in managing the condition. For more information about using Givlaari, see the package leaflet or contact your doctor or pharmacist.
The active substance in Givlaari, givosiran, is a synthetic small interfering RNA (a type of genetic material) that works by reducing the production of an enzyme involved in an early step of haem production in the liver. This prevents the build-up of the substances that cause the symptoms of the condition.
Givlaari was more effective than placebo (a dummy treatment) in reducing the yearly number of serious porphyria attacks. In a main study involving 94 patients, those who received Givlaari had on average 3 serious attacks of symptoms per year compared with 13 in those receiving placebo.
The most common side effects with Givlaari (which may affect more than 1 in 5 people) are reactions at the site of the injection, nausea (feeling sick) and tiredness. For the full list of side effects and restrictions, see the package leaflet.
A main study has shown that Givlaari is effective at reducing porphyria attacks. The side effects of Givlaari treatment were mostly mild and moderate and most resolved during the study.
The European Medicines Agency therefore decided that Givlaari’s benefits are greater than its risks and it can be authorised for use in the EU.
Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Givlaari have been included in the summary of product characteristics and the package leaflet.
As for all medicines, data on the use of Givlaari are continuously monitored. Side effects reported with Givlaari are carefully evaluated and any necessary action taken to protect patients.
Givlaari received a marketing authorisation valid throughout the EU on 2 March 2020.
Product information
Latest procedure affecting product information: II/0011/G
08/06/2023
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).
Product details
- Name of medicine
- Givlaari
- Active substance
- Givosiran
- International non-proprietary name (INN) or common name
- givosiran
- Therapeutic area (MeSH)
- Porphyrias, Hepatic
- Anatomical therapeutic chemical (ATC) code
- A16AX16
Pharmacotherapeutic group
Various alimentary tract and metabolism productsTherapeutic indication
Treatment of acute hepatic porphyria (AHP) in adults and adolescents aged 12 years and older.
News on Givlaari
Topics
This page was last updated on