https://www.gosh.nhs.uk/wards-and-departments/departments/clinical-specialties/dubowitz-neuromuscular-centre-dnc-information-parents-and-visitors/conditions-we-treat/givinostat-expanded-access-programme/

Givinostat Early Access Programme

This page was last updated 21 May 2025

This page is to provide patients and families with information regarding the Givinostat Early Access Programme (EAP) and accessing the drug as a patient at Great Ormond Street Hospital for Children.

We are currently contacting all patients who may be eligible for Givinostat. If your child may be eligible for the drug you should receive a letter in the post in the week commencing 26 May 2025.

What is the Givinostat Early Access Programme?

Givinostat is a potential treatment for Duchenne muscular dystrophy (DMD) that has been developed by the pharmaceutical company ITF Pharma LTD. This drug showed promise in clinical trials and is awaiting a decision from the National Institute of Clinical Excellence (NICE) as to whether it will be made available to patients on the NHS.

In the meantime, ITF Pharma Ltd have made the drug available to eligible patients through an Early Access Programme.

Givinostat EAP at GOSH

Whilst the manufacturer has made the drug available to NHS hospitals to give to their patients free of charge, there are significant additional costs to a hospital to enable us to safely administer the drug beyond the cost of the drug itself.

It has taken us some time to ensure we can cover these additional costs and have the staff we need in place to monitor the use of the drug in our patients. We understand how worrying this situation has been for patients and families and we are sorry for this.

We are pleased to confirm that we are now able to offer Givinostat to eligible patients in clinic at GOSH. Our clinical team are contacting all patients who may be eligible for the treatment to let them know it will be discussed in their next appointment.

We are working at pace and aim to see all potentially eligible patients within the next four months. We are currently prioritising those patients who require the treatment most urgently or are at risk of losing ambulation.

What is happening now?

Our clinical team are working through our patients and will be in touch with families directly when appointments are confirmed. The team are working incredibly hard to see patients at quickly as possible.

If you are a current GOSH patient and have any questions, please contact the neuromuscular CNS team at: neuromuscular.cns@gosh.nhs.uk

If you have any general questions, please contact our Patient Advice and Liaison service on email or phone 020 7829 7862.

Information for families

Which patients will be offered Givinostat?

The Early Access Programme for Givinostat is for ambulant patients only, and this means children who are able to walk or at least able to stand. In your child’s clinic appointment a member of the clinical team will discuss the eligibility criteria with you and how this relates to your child.

Can patients from other hospitals be referred to GOSH for treatment

We are only able to offer Givinostat to current GOSH patients. No referrals from other centres will be accepted.

When will my child’s appointment be?

We cannot say for certain when your child’s appointment will be at this stage, but we aim to see all potentially eligible patients in clinic in the next four months. We are prioritising patients who are at risk of losing ambulation – so those patients who can walk, but who cannot rise from the floor will be seen first.

I’m not sure if my child is eligible, what should I do?

We are sending letters to all patients with whom we will be discussing the possibility of them receiving Givinostat in their next appointment. Children who are most at risk of losing ambulation will be seen first.

I think my child might be eligible but I have not had a letter, what should I do?

We are currently sending letters to all patients who may be eligible and we expect them to receive these letters during the week commencing 26 May. If after this week you think you child meets the eligibility requirements for Givinostat and you have not had a letter from our clinical team please email our CNS team at: neuromuscular.cns@gosh.nhs.uk

Givinostat requires regular blood tests for monitoring purposes, will we have to come to GOSH for these?

We will make arrangements, where possible, with your local hospital for your child to have their monitoring blood tests there. It may be necessary for them to come to GOSH for further tests and we will discuss this with you at your child’s appointment.

What happens if NICE does not approve Givinostat for use in the NHS?

As part of the EAP the manufacturer has committed to continuing to supply the drug to patients who have already commenced treatment. This will be discussed with you in your appointment.

Will GOSH be joining the Givinostat EAP for non-ambulatory patients?

The current Givinostat EAP is for ambulatory patients only.