olipudase alfa at a glance
Medicine class
Enzyme replacement therapy
Main use
Non-central-nervous-system manifestations of ASMD type A/B or type B
Administration
Intravenous infusion prepared and administered by trained healthcare professionals
Important limitation
It is not expected to cross the blood–brain barrier or improve neurological manifestations
What is olipudase alfa used for?
Olipudase alfa is licensed as enzyme replacement therapy for:
Non-central-nervous-system manifestations of ASMD type A/B in children and adults.
Non-central-nervous-system manifestations of ASMD type B in children and adults.
How does olipudase alfa work?
People with ASMD have too little functioning acid sphingomyelinase, allowing sphingomyelin to accumulate in cells and damage organs. Olipudase alfa supplies a recombinant version of this enzyme, reducing sphingomyelin accumulation in affected organs. It does not reach the brain in clinically meaningful amounts.
olipudase alfa preparations and strengths
Solution for infusion
Route: Intravenous
Strengths: 20 mg
How to take olipudase alfa
Olipudase alfa is not swallowed. The powder is reconstituted, further diluted and given into a vein using a stepwise infusion under specialist supervision. Home infusion may be considered only after specialist assessment in someone who is stable and tolerating treatment, with trained personnel and emergency support available.
Report headache, hives, fever, nausea, vomiting, itching, breathing symptoms or other changes during the infusion immediately.
Do not alter a home-infusion procedure without the prescribing team's supervision.
olipudase alfa side effects
Common or expected effects
- Headache
- Hives, itching, rash or skin redness
- Fever
- Nausea or vomiting
- Abdominal pain
- Muscle aches
- Raised inflammatory markers
Get urgent medical advice
- Anaphylaxis or severe hypersensitivity, including breathing difficulty, wheezing, throat tightness or swelling beneath the skin
- Severe infusion-associated reactions
- Clinically important liver-enzyme elevation
- Fast or irregular heartbeat, particularly with faintness or low blood pressure
olipudase alfa in pregnancy
Human pregnancy experience is limited, and animal studies found reproductive toxicity. Use during pregnancy is not recommended unless the specialist judges that the potential maternal benefit outweighs the potential risks. Anyone pregnant, planning pregnancy or able to become pregnant should discuss treatment and contraception with the specialist team.
olipudase alfa while breastfeeding
It is unknown whether olipudase alfa enters human milk, and a risk to the infant cannot be excluded. The specialist and patient should jointly decide whether to pause breastfeeding or treatment after considering their respective benefits.
olipudase alfa interactions
Formal interaction studies have not been performed. Cytochrome P450-mediated interactions are not expected because olipudase alfa is a recombinant human protein, but the treatment team should still be told about all prescribed, non-prescription and complementary products.
Common questions about olipudase alfa
Answers are fully visible for fast scanning and source review.
What is olipudase alfa used to treat?
It treats manifestations outside the central nervous system in children and adults with ASMD type A/B or type B.
Is olipudase alfa an enzyme replacement therapy?
Yes. It supplies recombinant acid sphingomyelinase to replace the deficient enzyme in ASMD.
Does olipudase alfa treat the neurological effects of ASMD?
No. It is not expected to cross the blood–brain barrier, so it is not intended to modify central nervous system manifestations.
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.