Givinostat Expanded Access Programme

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

We recognise that this is a developing situation, and we will continue to keep this page updated.

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 Expanded Access Programme.

The manufacturer has made the drug available to NHS hospitals to give to their patients free of charge. However, there are significant additional costs to a hospital to enable us to safely administer the drug beyond the cost of the drug itself and these costs are not currently covered by the manufacturer.

For a drug such as Givinostat, patients require regular blood tests to ensure they are responding well to the treatment and not experiencing any side effects. Patients also require regular appointments and monitoring by our nursing teams.

This additional staff time and material costs are not something we can currently afford to provide at a time when our resources are more stretched than ever. In addition, GOSH – as a specialist centre for DMD, has a significantly greater number of patients than other hospitals – and subsequently the costs to us of rolling out Givinostat are much higher.

We recognise how encouraging the results from the Givinostat trial were and we are therefore committed to finding a solution which would allow our patients to benefit from the drug. We are working with our colleagues in both the North Star DMD clinical network and other children’s hospitals through the Children’s Hospital Alliance to fully understand the cost to hospitals in offering Givinostat, and to then identify ways to bridge this gap.

We understand how distressing this situation is to patients and families and want to assure you that we are working at pace to find a solution.

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

For more information on the Givinostat EAP please visit the following links:

• Muscular Dystrophy UK website
• Duchenne UK website