patisiran sodium at a glance
Medicine class
Small interfering RNA therapy
Licensed use
Hereditary transthyretin-mediated amyloidosis with stage 1 or stage 2 polyneuropathy in adults
Administration
A diluted intravenous infusion given by a healthcare professional after premedication
Key precaution
Treatment lowers blood vitamin A levels, so supplementation and assessment of new eye symptoms are important
What is patisiran sodium used for?
The selected UK product is licensed for:
Treating hereditary transthyretin-mediated amyloidosis in adults with stage 1 or stage 2 polyneuropathy.
How does patisiran sodium work?
Patisiran is carried in lipid nanoparticles to liver cells. It uses the natural RNA-interference pathway to break down messenger RNA for transthyretin, reducing production of both altered and normal transthyretin protein.
patisiran sodium preparations and strengths
Solution for infusion
Route: Intravenous
Strengths: 2 mg/mL
How to take patisiran sodium
Patisiran is not taken by mouth. The concentrate is prepared, diluted and infused into a vein by a healthcare professional.
Premedication is given beforehand to reduce infusion-related reactions.
The infusion site and the patient are observed for reactions; the infusion may be slowed or stopped if symptoms occur.
Home infusion may be considered after clinic infusions have been well tolerated and the treating specialist recommends it; a healthcare professional must administer it.
Take only the vitamin A supplementation advised by the specialist team.
patisiran sodium side effects
Common or expected effects
- Swelling of the hands, ankles or feet
- Infusion-related reactions, which can include flushing, nausea, pain, headache, rash, dizziness or breathing symptoms
- Bronchitis, sinusitis or rhinitis
- Vertigo
- Indigestion
- Joint pain or muscle spasms
- Skin redness
Get urgent medical advice
- A severe infusion or allergic reaction causing breathing difficulty, chest symptoms, facial swelling, marked dizziness, fainting or collapse
- New night blindness, persistent dry eyes, eye inflammation or other significant vision changes suggesting vitamin A deficiency
- Pain, swelling or leakage around the infusion site
patisiran sodium in pregnancy
Pregnancy should be excluded before treatment and effective contraception used during treatment. Patisiran is generally avoided during pregnancy because treatment disrupts vitamin A levels, which may affect fetal development. Anyone planning a pregnancy or becoming pregnant should contact the specialist promptly because treatment, supplementation and monitoring require specialist review.
patisiran sodium while breastfeeding
It is unknown whether patisiran enters human milk, and risk to a breastfed child cannot be excluded. The specialist and patient should decide whether to stop breastfeeding or withhold treatment after considering the benefits of each.
patisiran sodium interactions
Formal clinical interaction studies are limited. Tell the specialist team about all medicines and supplements.
Bupropion or efavirenz
An uncertain effect involving the CYP2B6 enzyme was seen in laboratory studies, so concomitant treatment should be reviewed.
Vitamin A supplements
Use only the amount directed by the specialist; blood vitamin A results during treatment do not reliably represent total body vitamin A.
Other cytochrome P450 substrates, inhibitors or inducers
Clinically important interactions are not generally expected because patisiran is not a cytochrome P450 substrate.
Common questions about patisiran sodium
Answers are fully visible for fast scanning and source review.
What is patisiran sodium used to treat?
It is licensed for adults with hereditary transthyretin-mediated amyloidosis who have stage 1 or stage 2 polyneuropathy.
Is patisiran sodium chemotherapy?
No. It is a small interfering RNA therapy that reduces production of transthyretin protein in liver cells.
How is patisiran sodium administered?
A healthcare professional filters and dilutes the concentrate before giving it as an intravenous infusion.
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.