Early Access Programme for givinostat now open in UK
In a major development for some Duchenne families, an Early Access Programme (EAP) for a new treatment for Duchenne muscular dystrophy is now open in the UK.
Summary
Givinostat is not yet approved in the UK, but the company is making it available free of charge in the UK until the regulators make a decision about approval. Only certain boys will be able to take part in the EAP. We have listed the criteria below.
If you think your son meets the criteria, you can only access it through your local neuromuscular clinic. It is up to YOU to contact your neuromuscular team and ask for your son to take part in the EAP. Some clinicians and NHS Trusts may decide not to take part in the EAP. You can only be treated at the clinic at which your son is registered.
If your NHS trust is not taking part, we urge you to contact the CEO of the Trust and explain your situation. We have written draft letters for you to use. If you do not get a satisfactory response from the Trust, let us know and we will help you contact your local MP.
Please know that Duchenne UK is committed to helping as many families as possible to take part in this scheme and we will be writing to the CEOs of all 24 NorthStar in the coming days sites to explain the situation.
If you have any questions, or need our help, please email w[email protected].
Who can take part in the EAP?
Your son can only take part in the EAP if he meets the following criteria. There are also some criteria below which will exclude you from taking part. Children who have previously been on gene therapy trials that are no longer active, are eligible.
– Confirmed diagnosis of DMD
– Age 6 and above, and ambulant
– On stable corticosteroid for at least 6 months prior to start the treatment
- Time to stand up: less than 10s
- Patient is not a candidate for any licensed or standard-of-care DMD therapy option -except for Corticosteroids- available at the time of inclusion
- Patient is not eligible for any ongoing clinical trial for DMD
– Patient must be willing to use adequate contraception
- Residing lawfully in the UK on a properly settled basis.
What is givinostat?
Givinostat is not a cure for DMD, though it may help preserve muscle function and slow down disease progression. It is an ‘HDAC inhibitor’. It blocks enzymes called histone deacetylases (HDACs), which are involved in turning genes ‘on’ and ‘off’ within cells. It works by targeting pathogenic processes to reduce inflammation and muscle loss. You can read a more technical explanation of givinostat by Professor Annemieke Aartsma-Rus on the World Duchenne Organisation’s website here.
Is givinostat approved in the UK?
Givinostat is not approved for DMD in the UK. Some boys in the UK took part in givinostat clinical trials, and it is currently being assessed by Britain’s regulators: the Medicines and Healthcare products Regulatory Agency (MHRA), and the National Institute for Health and Care Excellence (NICE), and the Scottish Medicines Consortium (SMC).
Givinostat was approved by the United States Food and Drug Administration in March 2024 for people with all genetic variants of DMD and who are six years of age and older. It is being considered by the MHRA through a new and faster pathway, called the International Recognition Procedure, with a decision expected in early 2025. While Duchenne UK welcome the use of this quicker procedure, considering givinostat is already licensed in the US we have been pushing for a faster decision. We wrote about it in the Daily Mail and you can read the article here.
NICE is also appraising givinostat, with a committee meeting scheduled for May 2025. If NICE recommend givinostat, then it will be available through the NHS in England, Wales and Northern Ireland. The SMC will assess givinostat in Scotland.
How do I access the EAP?
You can only access the EAP through your neuromuscular consultant, as only they can make a request for givinostat from the company. Decisions are made on a case-by-case basis for individual named patients. If you think you are eligible for givinostat, contact your clinician, and ask to discuss with them whether it would be appropriate for you.
If, for whatever reason, an NHS Trust decides to not take part in the EAP, your clinician will not be able to access the EAP – even if they would prefer to.
The EAP is only available in the following countries; Belgium, France, Germany, Italy, the Netherlands, Spain, and the UK.
What should I do if my doctor, or my local NHS Trust, does not want to take part in the EAP?
Duchenne UK believes that the clinicians treating our community are best placed to decide what will benefit patients medically, and we respect their opinions. But we do not consider other reasons to not take part in the EAP to be legitimate, for instance concerns over resourcing, logistics, staffing, or administrative burden. These should not be barriers to accessing medicine.
If you believe your doctor, or your local NHS Trust, is not taking part in the EAP for those kinds of reasons, we urge you to write to them – and to let us know so we can support you. You can find a template letter you can send to the CEO of your local NHS Trust here, and you can message us at [email protected].
Duchenne UK will be writing to the CEOs in charge of all the Trusts which have doctors who are part of the NorthStar Clinical Network. We will tell the CEOs we believe this treatment should be made available for people with DMD, request the Trust provides adequate support for their clinician if they wish to access the EAP, and offer to help them if we can. We know every Trust will be facing different challenges, and while some sites will have no problem accessing the EAP, others may struggle.
What is the Early Access Programme (EAP)?
ITF Pharma UK, the company behind givinostat, is providing access to their treatment early, before official approval, through a type of compassionate use scheme called an Early Access Programme (EAP). This is very different from another type of compassionate use scheme you may have heard of which Duchenne UK successfully campaigned for in 2014, called the Early Access to Medicines Scheme (EAMS).
Unlike EAMS, the EAP does not involve the MHRA nor NICE. There is no central approval within the NHS for the EAP, so the company must work with and sign contracts with each NHS Trust which wants to take part separately.
Who is, and is not, eligible for the EAP?
To be eligible for the EAP, the person with DMD must meet all the criteria below;
- Confirmed diagnosis of DMD,
– Age 6 and above, and ambulant,
– On stable corticosteroid for at least 6 months prior to start the treatment,
- Time to stand up in less than 10s,
- Patient is not a candidate for any licensed or standard-of-care DMD therapy option -except for Corticosteroids- available at the time of inclusion,
– Patient is not eligible for any ongoing clinical trial for DMD,
- Patient must be willing to use adequate contraception,
- Residing lawfully in the UK on a properly settled basis.
Any of the below criteria will exclude the person with DMD from the EAP;
- Patient registered for another EAP
– Use of any current DMD investigational drug
- Patient is participating in any ongoing givinostat clinical trial
- Patient is participating in an ongoing other clinical trial
- Have platelets count, at < Lower Limit of Normal (LLN)
- Have Triglycerides > 300 mg/dL (3.42 mmol/L) in fasting condition
- Patients who are at an increased risk for ventricular arrhythmias / concomitant use with other drugs that prolong the QTc interval
- Symptomatic cardiomyopathy or heart failure and/or left ventricular ejection fraction <45%
- Have any hypersensitivity to the components of the EAP medication
- Have a sorbitol intolerance or sorbitol malabsorption or have the hereditary form of fructose intolerance.