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 on 29 August 2016.
Givlaari contains the active substance Givosiran.
Givlaari is given by injection under the skin once a month. The dose depends on the patient’s weight.
The active substance in Givlaari, Givosiran, is a synthetic small interfering RNA 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 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 and tiredness.
A main study has shown that the side effects of Givlaari treatment were mostly mild and moderate and most resolved during the study.
The European Medicines Agency ( EMA ) therefore decided that Givlaari’s benefits are greater than its risks and it can be authorised for use in the EU. ( Xagena )
Source: EMA, 2020