Basic Information
EMA regulatory identification and product classification information
EMA Identifiers
Special Designations
EMA special regulatory designations and classifications
Overview Summary
Comprehensive product overview and regulatory summary
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 contains the active substance givosiran.
Active Substances (1)
Givosiran
Documents (14)
Givlaari : EPAR - Public assessment report
March 9, 2020
CHANGES_SINCE_INITIAL_AUTHORISATION
Givlaari : Orphan maintenance assessment report (initial authorisation)
March 9, 2020
CHANGES_SINCE_INITIAL_AUTHORISATION
Givlaari : EPAR - Procedural steps taken and scientific information after the authorisation
May 8, 2025
CHANGES_SINCE_INITIAL_AUTHORISATION
Givlaari : EPAR - Procedural steps taken and scientific information after the authorisation (archive)
March 18, 2021
CHANGES_SINCE_INITIAL_AUTHORISATION
CHMP summary of positive opinion for Givlaari
January 31, 2020
CHANGES_SINCE_INITIAL_AUTHORISATION
Givlaari : EPAR - All authorised presentations
March 9, 2020
AUTHORISED_PRESENTATIONS
CHMP summary of positive opinion for Givlaari
January 31, 2020
INITIAL_MARKETING_AUTHORISATION_DOCUMENTS
Givlaari : EPAR - Product information
March 9, 2020
DRUG_PRODUCT_INFORMATION
Givlaari : EPAR - Medicine overview
March 9, 2020
OVERVIEW_DOCUMENT
Givlaari : EPAR - Procedural steps taken and scientific information after the authorisation
March 18, 2021
CHANGES_SINCE_INITIAL_AUTHORISATION
Givlaari : EPAR - Public assessment report
March 9, 2020
INITIAL_MARKETING_AUTHORISATION_DOCUMENTS
Givlaari : Orphan maintenance assessment report (initial authorisation)
March 9, 2020
INITIAL_MARKETING_AUTHORISATION_DOCUMENTS
Givlaari-PSUSA-00010839-202011 : EPAR - Scientific conclusions and grounds for the variation to the terms of the marketing authorisation(s)
October 12, 2021
CHANGES_SINCE_INITIAL_AUTHORISATION
Givlaari : EPAR - Risk-management-plan summary
March 9, 2020
RISK_MANAGEMENT_PLAN_SUMMARY
Overview Q&A (7)
Question
What are the risks associated with Givlaari?
Answer
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.
Question
What measures are being taken to ensure the safe and effective use of Givlaari?
Answer
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.
Question
Why is Givlaari authorised in the EU?
Answer
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.
Question
Other information about Givlaari
Answer
Givlaari received a marketing authorisation valid throughout the EU on 2 March 2020.
Question
How is Givlaari used?
Answer
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.
Question
How does Givlaari work?
Answer
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.
Question
What benefits of Givlaari have been shown in studies?
Answer
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.