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Givlaari
Australian Prescription Medicine Decision Summary
Published
Product name
Givlaari
Active ingredient
Givosiran
Submission type
New chemical entity
Decision
Approved
Decision date
Registration date
What this medicine was approved for
Givlaari (givosiran) was approved for the following therapeutic use:
Givlaari is indicated for the treatment of acute hepatic porphyria (AHP) in adults and adolescents aged 12 years and older.
How this medicine works
Givosiran is a double-stranded small interfering ribonucleic acid (siRNA) that causes degradation of aminolevulinic acid synthase 1 (ALAS1) messenger ribonucleic acid (mRNA) in hepatocytes through RNA interference. ALAS1 is the first and rate-limiting enzyme of heme synthesis in the liver. Its expression is induced in AHP due to a loss-of-function gene mutation in a downstream heme synthesis enzyme. Givosiran acts to reduce elevated levels of liver ALAS1 mRNA. This leads to reduced circulating levels of neurotoxic heme intermediates aminolevulinic acid and porphobilinogen, the key causal factors of attacks and other disease manifestations of AHP.
Why the TGA approved or did not approve this medicine
The decision was based on quality (chemistry and manufacturing), nonclinical (pharmacology and toxicology), clinical (pharmacology, safety and efficacy) and risk management plan information submitted by the sponsor. The benefit-risk profile of Givlaari was considered favourable for the therapeutic use approved.
Sponsor