Human medicines European public assessment report (EPAR): Givlaari, givosiran, Porphyrias, Hepatic, Date of authorisation: 02/03/2020, Revision: 5, Status: Authorised
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.
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