OAV101-IT, a potential new gene therapy for SMA, not yet recommended for NHS use in England

29 July 2026

OAV101-IT could give more people with spinal muscular atrophy (SMA) access to a one-time gene therapy treatment. Further evidence will now be considered before a final recommendation is made about routine NHS use in England.

The National Institute for Health and Care Excellence (NICE) is the organisation responsible for deciding whether treatments should be funded by the NHS in England. NICE is currently assessing whether OAV101-IT should be made available for people aged two years and over living with SMA.

At its first committee meeting, NICE concluded that there was still too much uncertainty in the evidence to determine whether OAV101-IT represents good value for money for the NHS. However, this does not mean the treatment has been rejected.

There is now an opportunity for the company that makes the treatment, clinicians, patient organisations and the public to respond to the committee’s feedback and submit further evidence before a final recommendation is made. While this news may be disappointing for the SMA community, it is important to remember that this is not the final decision. Treatments are often not recommended at this stage of the NICE process, and OAV101-IT will be considered again once further evidence has been reviewed.

What is OAV101-IT?

OAV101-IT targets the genetic cause of SMA by delivering a functional copy of the SMN1 (survival motor neuron) gene into nerve cells. It has the same active ingredient as Zolgensma (onasemnogene abeparvovec), an approved treatment for some babies and young children with SMA. However, OAV101-IT is administered differently.

While Zolgensma is given as a one-time infusion into the bloodstream, OAV101-IT is given as a one-time injection into the fluid surrounding the spinal cord (an intrathecal injection, also known as a lumbar puncture).

This approach is designed to deliver the treatment directly to the central nervous system, where the motor neurons affected by SMA are located. The intrathecal route may allow the treatment to be used in a broader range of people with SMA, including older children and adults who are not eligible for treatment with Zolgensma.

Why did the committee make this decision?

The committee identified several areas of uncertainty that it felt needed further exploration before a final decision could be made. These included:

• Who might receive the treatment and when – OAV101-IT has not been tested in adults, so the committee wants to better understand who might choose to switch from other treatments (such as nusinersen or risdiplam) to OAV101-IT and when.
• Whether people might receive additional treatment after OAV101-IT – Clinical experts expect that most people who receive OAV101-IT would not need other treatments (such as nusinersen or risdiplam). This would only be expected in rare cases where OAV101-IT does not provide the expected clinical benefit. The committee wants to understand whether this reflects what is likely to happen in practice and how people living with SMA would feel about this approach.
• The current experience of existing treatments – Some people living with SMA choose to stop, or need to stop, treatment with nusinersen or risdiplam. The committee wants to understand how often this happens in practice and whether the evidence used in the assessment reflects real-world experience.

Can I access OAV101-IT?

No, OAV101-IT is not currently available in the UK.

Before a treatment can become routinely available on the NHS, it must be reviewed by the Medicines and Healthcare products Regulatory Agency (MHRA), which assesses whether it is safe and effective. It must also be assessed by organisations that decide whether it should be funded by the NHS.

OAV101-IT is currently being reviewed by the MHRA. In England, NHS funding decisions are made by NICE, which has not yet made a final decision on OAV101-IT. Wales and Northern Ireland generally follow NICE guidance, while Scotland has its own decision-making body, the Scottish Medicines Consortium (SMC). OAV101-IT has not yet been submitted to the SMC for assessment.

How can I get involved?

The consultation period is an opportunity for the SMA community to help inform NICE’s final decision on OAV101-IT.

We will be submitting comments to NICE, alongside SMA UK, to highlight the experiences and priorities of people living with SMA. We are particularly interested in hearing about the issues raised by the committee, as well as wider experiences that may help NICE understand the impact of SMA treatments in everyday life, including:

• what maintaining stability means to you and how this affects your daily life
• your experiences of starting, continuing, switching or stopping SMA treatments
• the factors that might influence a decision to switch to OAV101-IT
• what access to a one-time gene therapy treatment could mean for you or your family compared with lifelong treatment

If you would like to share your views, please get in touch with us at campaigns@musculardystrophyuk.org

Anyone can also submit comments directly to NICE before the consultation closes on 19 August. Details of how to respond can be found on the NICE website.