This page is to provide patients and families with information regarding the givinostat Early Access Programme and accessing the drug as a patient at Evelina London Children’s Hospital.
We will be sending letters to the families of all existing Evelina London patients who may be eligible for givinostat in the next few weeks.
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 medicine showed promise 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 Ltd have made the decision to fund the medicine for eligible patients through an Early Access Programme.
Givinostat Early Access Programme at Evelina London Children’s Hospital
Whilst the manufacturer has made the medicine available free of charge for NHS hospitals to give to their patients, there are significant additional clinical resource costs to a hospital to enable it to be given to patients safely.
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 medicine. We know this has been a difficult and worrying situation for patients and families, and we have been working quickly to identify solutions.
We are pleased to confirm that we are now able to offer givinostat to eligible children with Duchenne muscular dystrophy in our clinic at Evelina London Children’s Hospital.
We are working at pace and aim to see and review all potentially eligible patients in the coming months. We are currently prioritising children who require the treatment most urgently or are at a higher risk of losing movement (ambulation) soon.
What is happening now?
Our clinical team are working through our patients and will be in touch with families who may be eligible to receive givinostat through the Early Access Programme.
Who is eligible?
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.
Can patients from other hospitals be referred to Evelina London Children's Hospital for treatment
We are only able to offer givinostat to current Evelina London Children’s Hospital patients. Unfortunately, we will not be able to accept referrals from other hospitals.
When will appointments start?
We cannot say for certain when appointments will be at this stage. 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.
Letters and further information are being sent to all potentially eligible families in the next few weeks.
Questions?
If your child is a current Evelina London Children’s Hospital patient and you have any questions, please email EvelinaNeuroCNS@gstt.nhs.uk