givosiran sodium at a glance
Medicine class
Liver-targeted small interfering RNA therapy
Licensed use
Acute hepatic porphyria in adults and adolescents aged 12 years and over
Administration
Injection under the skin, given by a healthcare professional
Key monitoring
Liver function, kidney function and blood homocysteine
What is givosiran sodium used for?
Licensed for:
Treatment of acute hepatic porphyria in adults and adolescents aged 12 years and over.
How does givosiran sodium work?
Givosiran uses RNA interference in liver cells to break down messenger RNA for the enzyme ALAS1. This reduces production of aminolevulinic acid and porphobilinogen, neurotoxic haem precursors that contribute to attacks and other manifestations.
How to take givosiran sodium
A doctor or nurse injects the ready-to-use solution under the skin.
Injection sites include the abdomen, upper arm or thigh.
Sites should be rotated.
It should not be injected into scarred, red, inflamed or swollen skin.
The vial is for single use and should be used immediately after opening.
givosiran sodium side effects
Common or expected effects
- Nausea
- Redness, pain, itching or swelling at the injection site
- Rash, itching, eczema or hives
- Tiredness
- Raised liver enzymes
- Increased creatinine or reduced kidney filtration
- Raised blood homocysteine
Get urgent medical advice
- Anaphylaxis or another severe hypersensitivity reaction, including facial or throat swelling, breathing difficulty, severe dizziness or fainting
givosiran sodium in pregnancy
Human pregnancy information is limited. Treatment may be considered only after specialist assessment of expected benefit and potential fetal risk.
givosiran sodium while breastfeeding
It is unknown whether givosiran enters human milk, and risk to a breastfed child cannot be excluded. The specialist team should weigh the benefits of breastfeeding and treatment when deciding whether either should be paused.
givosiran sodium interactions
Givosiran can reduce activity of some liver enzymes and increase exposure to certain medicines.
CYP1A2 substrate medicines
Their effects or adverse effects may be increased or prolonged; treatment should be reviewed and adjusted where appropriate.
CYP2D6 substrate medicines
Their effects or adverse effects may be increased or prolonged, particularly for medicines with a narrow therapeutic range.
Common questions about givosiran sodium
Answers are fully visible for fast scanning and source review.
Does givosiran sodium cure acute hepatic porphyria?
No. It does not correct the inherited enzyme defect; it reduces liver production of harmful haem precursors by silencing ALAS1 messenger RNA.
Is it used to stop an attack that has already started?
It is intended to reduce ongoing disease activity and future attacks rather than act as an on-demand painkiller or emergency treatment.
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.