pegunigalsidase alfa at a glance
Medicine class
Pegylated recombinant enzyme replacement therapy
Licensed use
Long-term treatment of adults with confirmed Fabry disease
Administration
Intravenous infusion prepared and given under specialist supervision
Home treatment
May be considered for medically stable people who have tolerated infusions well, with suitable training and support
What is pegunigalsidase alfa used for?
The licensed use is:
Long-term enzyme replacement therapy in adults with confirmed Fabry disease caused by alpha-galactosidase deficiency.
How does pegunigalsidase alfa work?
Fabry disease causes reduced alpha-galactosidase A activity, allowing Gb3 and lyso-Gb3 to accumulate in cells. Pegunigalsidase alfa is a pegylated recombinant form of this enzyme that enters lysosomes and helps break down these substances.
pegunigalsidase alfa preparations and strengths
Solution for infusion
Route: Intravenous
Strengths: 2 mg/mL
How to take pegunigalsidase alfa
It is diluted and given into a vein by a trained healthcare professional. It must not share an intravenous line with other products and is given through a low-protein-binding filter. People are observed for infusion reactions afterwards. Home administration requires specialist assessment, training and ongoing clinical support.
Do not shake the vials or prepared infusion.
Report symptoms developing during or soon after an infusion immediately.
Do not drive or operate machinery if dizziness, vertigo or fainting occurs after treatment.
pegunigalsidase alfa side effects
Common or expected effects
- Infusion-related reactions
- Nausea, abdominal pain, diarrhoea or vomiting
- Headache, dizziness or vertigo
- Weakness or fatigue
- Rash, itching or skin redness
- Muscle or joint pain
- Chills
- Tingling or pins and needles
- Chest discomfort or pain
Get urgent medical advice
- Severe hypersensitivity or anaphylaxis, including facial or throat swelling, bronchospasm, wheezing, breathing difficulty, low blood pressure or collapse
- New or worsening protein in the urine or renal impairment, which may rarely indicate an immune-complex kidney reaction
pegunigalsidase alfa in pregnancy
Human pregnancy data are absent or very limited. Pegunigalsidase alfa should be avoided during pregnancy unless clearly necessary; discuss pregnancy or plans for pregnancy with the specialist team.
pegunigalsidase alfa while breastfeeding
It is unknown whether pegunigalsidase alfa passes into human milk, and a risk to a breastfed infant cannot be excluded. The specialist team should weigh the benefits of breastfeeding against the benefits of continuing treatment.
pegunigalsidase alfa interactions
No formal interaction studies have been performed. Tell the specialist team about all prescribed, non-prescribed and complementary products.
Medicines metabolised by cytochrome P450 enzymes
A metabolic interaction is considered unlikely because pegunigalsidase alfa is a protein expected to be broken down by peptide hydrolysis.
Common questions about pegunigalsidase alfa
Answers are fully visible for fast scanning and source review.
What is pegunigalsidase alfa used for?
It is licensed for long-term enzyme replacement therapy in adults with confirmed Fabry disease.
Is pegunigalsidase alfa an enzyme replacement therapy?
Yes. It replaces or supplements the deficient alpha-galactosidase A enzyme in Fabry disease.
How is pegunigalsidase alfa given?
It is diluted and administered as an intravenous infusion under specialist supervision.
Can pegunigalsidase alfa be given at home?
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.