The human medicines committee of the European Medicines Agency has recommended granting a European Union (EU) licence to givosiran (Givlaari), the first treatment for acute hepatic porphyria (AHP) in adults and adolescents aged 12 years and older.
Givosiran is a synthetic double-stranded small interfering ribonucleic acid agent (siRNA) oligonucleotide directed against delta-aminolevulinic acid synthase 1 mRNA (ALAS1), which alleviates the build-up of liver ALAS1 mRNA and reduces levels of the neurotoxic intermediates aminolevulinic acid (ALA) and porphobilinogen (PBG).
The benefits and safety of givosiran were demonstrated in a phase 3 clinical study involving 94 patients with AHP who experienced at least two attacks in the past six months. Data from the study showed that givosiran was associated with a significant decrease in annual attacks and pain levels, and improved quality of life.
The most common side effects were injection site reactions, nausea, fatigue and abnormalities in kidney and liver function.
Givosiran will be available as 189 mg/ml solution for injection. Detailed recommendations on its use will be published once the licence has been granted by the European Commission.