Givinostat Early Access Programme (EAP)

This page is to provide patients and families with information about the Givinostat Early Access Programme and accessing the medicine as a patient at Bristol Royal Hospital for Children (BRHC).

We have started contacting families of patients at BRHC who may be eligible for Givinostat.

What is the Givinostat EAP?

Givinostat is a potential treatment for Duchenne muscular dystrophy (DMD) that has been developed by the pharmaceutical company ITF Pharma Limited.

This medicine had some positive results in clinical trials and is awaiting a decision from the National Institute of Heath and Care Excellence (NICE) as to whether it will be made available to patients on the NHS.

In the meantime, ITF Pharma Limited have made the decision to fund the medicine for eligible patients through an early access programme.

Givinostat EAP at BRHC

While the manufacturer has made the medicine available free of charge for this programme, there are significant extra costs to us to enable it to be given to patients safely.

It has taken us some time to make sure we can cover these costs and have the staff in place to monitor the use of the medicine. We know this has been a difficult and worrying situation for patients and families and we have been working quickly to resolve this.

We can confirm that UHBW is participating in the Givinostat EAP.

Which patients will be offered Givinostat?

The Givinostat EAP is for ambulant patients only. This means children who can walk or at least stand.

What is happening now?

Our clinical team is now carefully reviewing our patient list to see who is clinically eligible for treatment, and prioritising based on greatest clinical need.

If your child is eligible, we will be in touch directly to discuss the next steps.