Givlaari (givosiran) has been approved by Swissmedic for the treatment of acute hepatic porphyria (AHP) in adults and adolescents aged 12 years and older. This authorization provides a new therapeutic option for patients suffering from this rare and debilitating genetic disease.
AHP is characterized by a defect in one of the proteins responsible for heme production in the liver, leading to the accumulation of aminolevulinic acid (ALA) and porphobilinogen (PBG). Excessive levels of these substances can cause nerve damage, resulting in severe attacks with symptoms such as pain, nausea, muscle weakness, and cognitive dysfunction. Long-term complications can include hypertension, chronic kidney disease, and liver disease.
Givosiran, the active substance in Givlaari, works by reducing the amount of ALAS1, an enzyme responsible for ALA and PBG production in the liver. By decreasing ALAS1 levels, givosiran reduces the production of ALA and PBG, thereby mitigating the effects of AHP. Givlaari has been designated as an orphan drug due to the rarity and life-threatening nature of AHP.
Swissmedic's decision to authorize Givlaari was based on Article 13 of the Therapeutic Products Act (TPA), which allows for the consideration of assessments made by foreign regulatory agencies with comparable medicinal product control. In this case, Swissmedic accepted the assessment and approval decision of the European Medicines Agency (EMA) and conducted a limited scientific review.
Further details regarding Givlaari, including information for healthcare professionals and patients, can be found in the Swiss Public Assessment Report (SwissPAR) and related documents.
