Update on givinostat – and what it means for families

16 March 2026

The decision on whether Duchenne muscular dystrophy treatment, givinostat, can be offered on the NHS in England has been on pause since late 2025, and that pause is now continuing. We know this delay is frustrating, but there are encouraging signs: final discussions are progressing, and the review is now waiting for new rules in April that could help more treatments be approved. We are hopeful this will lead to a positive outcome.

Givinostat has been looked at by the National Institute for Health and Care Excellence (NICE) for more than a year. NICE decides which treatments can be used on the NHS in England, and its decisions are usually followed in Wales and Northern Ireland. Although givinostat was approved for NHS use in Scotland in December 2025, families in the rest of the UK are still waiting for NICE’s final decision.

Why the delay?

In late 2025, NICE announced they had paused the review to allow the company who makes givinostat, ITF Pharma UK, and NHS England to negotiate the price of the treatment. These discussions are still ongoing and need more time, which is why the pause remains in place.

We know this is incredibly frustrating and means continued uncertainty about long term access. But the latest update suggests encouraging progress behind the scenes.

Final discussions between ITF Pharma UK and NHS England are ongoing, and the pause gives the time needed for these conversations to reach a conclusion. It also allows the review to restart under updated NICE rules coming in April, which are designed to give more treatments a chance of being approved.

Taken together, these developments mean that givinostat is in a stronger position than it was a few months ago. Although we cannot predict the final decision, we are hopeful that these changes will support a positive outcome.

What are the new NICE rules?

NICE is updating the way it judges whether treatments offer good value for the NHS. This includes increasing the threshold it uses when deciding if a treatment provides enough benefit for its cost. The change is expected to help more treatments be approved than before, especially those that were very close to meeting the previous criteria.

The new rules are expected to come into effect in April, once they are approved by government. However, there is still some uncertainty about exact timelines. This means we don’t yet know when the review will restart or when the next update will be available. We will let you know as soon as we hear more.

What this means for families right now

Some boys and young men have been able to receive givinostat through an Expanded Access Programme (EAP). Through the EAP, ITF Pharma UK has been supplying the treatment to the NHS free of charge.

Families already receiving the treatment through the EAP will continue to do so. The EAP should also stay open for new families to start treatment until NICE gives its final decision.

However, not all centres have been able to take part because the EAP does not fund the staff time or resources needed to deliver the treatment safely. These centres have said they need to wait until NICE’s final decision. This has created a postcode lottery, with some boys and young men able to start treatment and others unable to access it.

This situation is incredibly frustrating and deeply unfair for those unable to access givinostat through the EAP. Together with the charities Duchenne UK and Action Duchenne, we have been pushing ITF Pharma UK, NHS England and NICE throughout this pause to reach a solution as quickly as possible – one that allows givinostat to move into routine NHS use so that those who can benefit can access it.

We will keep pushing urgently until this becomes a reality.