onasemnogene abeparvovec at a glance
Medicine class
Gene replacement therapy using a non-replicating AAV9 vector
Usual use
Specified forms of 5q spinal muscular atrophy caused by bi-allelic SMN1 mutations
Administration
Intravenous infusion in a specialist clinical centre
Important monitoring
Liver function, blood counts, kidney function and cardiac troponin
What is onasemnogene abeparvovec used for?
UK-licensed uses are:
5q spinal muscular atrophy with a bi-allelic SMN1 mutation and a clinical diagnosis of SMA type 1.
5q spinal muscular atrophy with a bi-allelic SMN1 mutation and no more than three copies of the SMN2 gene.
How does onasemnogene abeparvovec work?
In 5q SMA, both copies of SMN1 are altered, so not enough survival motor neuron protein is made. A non-replicating AAV9 vector carries a functional SMN1 gene into cells, including motor neurons, allowing them to produce SMN protein. It cannot restore motor neurons already lost.
How to take onasemnogene abeparvovec
An experienced SMA team prepares and administers this treatment in a clinical centre.
It is infused into a vein using a syringe pump; it must not be given as an intravenous push or bolus.
The patient should be clinically stable. Active infection may require treatment to be postponed.
Caregivers should use gloves and careful hand hygiene when handling bodily waste. Double-bag disposable nappies before household disposal.
Do not donate blood, organs, tissues or cells after treatment.
onasemnogene abeparvovec side effects
Common or expected effects
- Raised liver enzymes
- Liver toxicity
- Vomiting
- Low platelet count
- Raised cardiac troponin
- Fever
Get urgent medical advice
- Acute liver injury or liver failure, sometimes fatal
- Thrombotic microangiopathy, involving low platelets, red-cell damage and possible acute kidney injury
- Anaphylaxis or another serious infusion-related reaction
onasemnogene abeparvovec in pregnancy
There are no human pregnancy data, and reproductive animal studies have not been performed. Actual or planned exposure during pregnancy requires specialist discussion because safety is unknown.
onasemnogene abeparvovec while breastfeeding
Human data during breastfeeding are unavailable. If breastfeeding is relevant, the specialist team should assess the uncertainties and individual circumstances.
onasemnogene abeparvovec interactions
Formal interaction studies have not been performed. Important practical considerations include:
Medicines or substances that can damage the liver
Experience is limited. The specialist team should review exposure because liver toxicity is an important treatment risk.
Other treatments targeting 5q SMA
Evidence for combined or sequential use is limited and requires specialist assessment.
Live vaccines
Live vaccines should not be given while the patient is receiving an immunosuppressive corticosteroid regimen. Vaccination timing should be coordinated with the treatment centre.
Common questions about onasemnogene abeparvovec
Answers are fully visible for fast scanning and source review.
What is onasemnogene abeparvovec used for?
It treats specified forms of 5q spinal muscular atrophy caused by bi-allelic SMN1 mutations.
How does it help spinal muscular atrophy?
It delivers a functional SMN1 gene so motor neurons can make more survival motor neuron protein.
Does it cure spinal muscular atrophy?
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.