Givosiran
Givosiran (pronounced as giv-oh-sir-an) is a medication used for the treatment of acute hepatic porphyria (AHP) in adults. It is a small interfering RNA (siRNA) that works by decreasing the levels of aminolevulinic acid synthase 1 (ALAS1) in the liver, thereby reducing the accumulation of neurotoxic intermediates aminolevulinic acid (ALA) and porphobilinogen (PBG).
Etymology
The name "Givosiran" is derived from the drug's mechanism of action. "Givo-" is a prefix used in the naming of drugs that are small interfering RNAs, and "-siran" is a suffix used for drugs that target the liver.
Pharmacology
Givosiran is a small interfering RNA (siRNA) that targets aminolevulinic acid synthase 1 (ALAS1), an enzyme in the liver that is involved in the production of heme. By reducing the activity of ALAS1, givosiran decreases the production of neurotoxic intermediates aminolevulinic acid (ALA) and porphobilinogen (PBG) that cause the symptoms of acute hepatic porphyria.
Clinical Use
Givosiran is used for the treatment of acute hepatic porphyria (AHP) in adults. AHP is a group of rare genetic disorders characterized by potentially life-threatening attacks and, for some patients, chronic debilitating symptoms that negatively impact daily functioning and quality of life.
Side Effects
Common side effects of givosiran include nausea, injection site reactions, and fatigue. Serious side effects may include liver problems, kidney problems, and allergic reactions.
Related Terms
External links
- Medical encyclopedia article on Givosiran
- Wikipedia's article - Givosiran
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