Intrathecal Onasemnogene Abeparvovec (OAV101 IT)
Intrathecal Onasemnogene Abeparvovec (OAV101 IT)
Last updated: 21st September 2026
What is intrathecal onasemnogene abeparvovec (OAV101 IT) ?
This is a new version of the gene therapy Zolgensma, for treating adult and paediatric patients aged 2 years and older with SMA. It is not currently avaiable through the NHS.
How is it different from Zolgensma?
Intrathecal onasemnogene abeparvovec (OAV101 IT) is a new version of the gene therapy Zolgensma for treating adult and paediatric patients aged 2 years or older with SMA.
Like zolgensma, it delivers a healthy copy of the SMN1 gene to motor neurons. However, instead of being given into the blood stream (intravenously), Intrathecal onasemnogene abeparvovec (OAV101 IT) is administered through injection into the fluid surrounding the brain via a single injection by lumbar puncture. The amount given doesn’t need to be adjusted for age or body weight in older patients.
As for all treatments for SMA, Intrathecal onasemnogene abeparvovec (OAV101 IT) works best if started before there is any muscle weakness, or when this is minimal. It is therefore important for treatment to be started as soon as possible.
As of 2nd July 2026, the European Commission approved the drug for the treatment of children two years and older, teen and adults living with 5q SMA in Europe. It has not yet been approved by the Medicines and Healthcare products Regulatory Agency (MHRA) in the UK and is not currently available through the NHS, however work towards this possibility has begun.
Will it be available in the UK?
The National Institute for Health and Care Excellence (NICE) is considering whether to recommend that NHS England funds this treatment for people aged 2 years and over.
NICE needs to decide if a new drug works well and is good value for money for the NHS. This process relies heavily on hearing about the daily reality of life with SMA.
SMA UK and our partner organisations will be working closely with the NICE appraisal committee to make sure the patient and carer perspective is clearly understood. As the process moves forward, SMA UK will be sharing opportunities for you to share your thoughts and experiences on the topic to inform the NICE committee.
Read more about our advocacy for this treatment >
We are not aware of there being any plans yet for Intrathecal onasemnogene abeparvovec (OAV101 IT) to be considered by the Scottish Medicines Consortium (SMC).