Advocacy for Access to Intrathecal Onasemnogene Abeparvovec (OAV101 IT)
Advocacy for Access to Intrathecal Onasemnogene Abeparvovec (OAV101 IT)
Last updated: 21st September 2026
NICE issues draft guidance about interim access to the Gene intrathecal onasemnogene abeparvovec (OAV101 IT)
Following the committee meeting held on 7th July 2026, the National Institute of Health and Care Excellence (NICE) has published draft guidance not to offer this treatment on the NHS in England. NICE was considering access for anyone aged 2 years or older with 5Q SMA who has never had intravenous (IV) Zolgensma.
This draft decision does not affect access to Intravenous Zolgensma
NICE’s have given these reasons for this interim decision:
- Not Enough Data for Older Ages: Studies showed short-term movement improvements in children aged 2 to 18. However, there was very little information for teenagers over 12, and no test results for adults 18 and older.
- Hard to Compare with Existing Medicines: Most people aged 2 and over in England already take regular treatments like nusinersen (Spinraza) or risdiplam (Evrysdi). Medical trials did not test the gene therapy directly against these other medicines.
- Uncertain Long-Term Effects: It’s not clear how long the benefits of the treatment last over a person’s whole life.
- Value for Money Concerns: Because key information is missing, they could not properly calculate whether the treatment provides good value for the NHS.
Value for Money Concerns: Because key information is missing, they could not properly calculate whether the treatment provides good value for the NHS.
This is not a final decision.
The next NICE committee meeting when NICE will consider all the feedback will be on 6th October 2026. The community’s Patient representatives and clinicians will take part in this meeting.
Registration to watch this committee meeting is now open on the NICE website. The meeting will be held virtually, using Zoom.
You can find the links to register directly on Zoom on this page.
Please read the ‘Important information on how to register’ carefully.
As you will be able to register directly on Zoom for the meeting, you will automatically be granted a place, so please note the meeting date and time in your diary as you will no longer be sent a confirmation email. Your joining link and further instructions will be sent to you 1-2 days before the meeting.
Registration will close at 4pm on Tuesday 29 September 2026.
If registering as a public observer, you will be able to listen to the business of the meeting, except where confidential information is being discussed. You will not be able to:
- participate in committee discussions
- ask questions, take part in voting or put your views to members of the committee
For further information about meetings in public please refer to the following document: Common questions and answers about virtual committee meetings.
See the NICE Website >
Read the full Draft Guidance >
Read more about intrathecal onasemnogene abeparvovec (OAV101 IT) >
Currently intrathecal onasemnogene abeparvovec (OAV101 IT) doesn’t have marketing authorisation within the UK. The UK Medicines and Healthcare products Regulatory Agency (MHRA) is looking at the application for this.
Intrathecal onasemnogene abeparvovec (OAV101 IT) was approved on 24th November 2025 by the USA Food and Drug Administration (FDA) to treat SMA in adult and paediatric patients 2 years of age and older with a confirmed mutation in SMN1.
On 2nd July 2026, the European Commission approved Intrathecal onasemnogene abeparvovec (OAV101 IT) for people aged two years and over living with 5q SMA.
Our Advocacy and Access Diary
NICE held its first committee meeting to consider all the evidence. Patient and Clinical Experts gave their testimonies and answered NICE’s questions.
Towards the end of 2025, NICE invited patient groups and clinicians to give their views on this possibility. Patient Groups responded jointly. We based what we said on the SMA Community’s responses to our surveys on access to treatments. These were carried out between 2024 and January 2026. Thank you to those of you who have given their views. We have seen how important they are and how NICE listens to them when we have taken part in other treatment reviews.