lumasiran sodium at a glance
Medicine class
Small interfering RNA therapy
Licensed use
Primary hyperoxaluria type 1 in all age groups
Administration
Subcutaneous injection given by a healthcare professional
Treatment setting
Started and supervised by a clinician experienced in hyperoxaluria
What is lumasiran sodium used for?
Licensed use:
Treatment of primary hyperoxaluria type 1 in adults and children of all ages.
How does lumasiran sodium work?
Lumasiran sodium uses RNA interference in liver cells to reduce glycolate oxidase production by targeting HAO1 messenger RNA. This lowers glyoxylate available for conversion into oxalate and reduces urinary and plasma oxalate. Its action does not depend on the particular AGXT mutation.
lumasiran sodium preparations and strengths
Injection
Route: Parenteral
Strengths: 94.5 mg/0.5 mL
How to take lumasiran sodium
Lumasiran sodium is a ready-to-use, single-use solution given under the skin by a healthcare professional.
Injection sites include the abdomen, upper arms and thighs.
Injection sites are rotated, avoiding scars or skin that is red, inflamed or swollen.
lumasiran sodium side effects
Common or expected effects
- Injection-site reactions, including redness, pain, itching, swelling, bruising, discomfort or skin-colour change.
- Abdominal pain or discomfort.
Get urgent medical advice
- Seek urgent help for signs of a severe allergic reaction, particularly breathing difficulty, throat or tongue swelling, fainting or collapse.
lumasiran sodium in pregnancy
Human pregnancy data are unavailable. Use may be considered after specialist review of expected benefit for the woman and potential uncertainty for the fetus.
lumasiran sodium while breastfeeding
It is unknown whether lumasiran or its metabolites pass into human milk, and risk to a breastfed child cannot be excluded. The benefits of breastfeeding and treatment should be weighed.
lumasiran sodium interactions
Clinical interaction studies have not been performed, but clinically important CYP-mediated interactions are not expected from available laboratory evidence.
Pyridoxine (vitamin B6)
Concomitant use did not meaningfully alter the pharmacokinetics or pharmacodynamic effect of lumasiran.
Medicines handled by cytochrome P450 enzymes
Lumasiran is not expected to inhibit or induce CYP enzymes, although dedicated clinical interaction studies are unavailable.
Common questions about lumasiran sodium
Answers are fully visible for fast scanning and source review.
Is lumasiran sodium licensed for children?
Yes. Its licence covers primary hyperoxaluria type 1 in all age groups, although clinical data are limited in children younger than one year.
Does lumasiran sodium treat every type of primary hyperoxaluria?
No. Its licence is specifically for primary hyperoxaluria type 1.
Is lumasiran sodium a gene therapy?
It is an RNA-interference treatment that temporarily reduces translation of HAO1 messenger RNA; it does not edit or replace genes.
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.