Givinostat – our role in supporting access to new treatments
2 May 2025
Researchers are finally starting to find treatments which slow progression for people with muscle wasting and weakening conditions. Promising treatments, like givinostat for Duchenne muscular dystrophy, are now within reach – but not enough people can access them. We’re working tirelessly to change this, to ensure everyone gets the treatment they need, when they need it.
In the UK, before a new treatment can be prescribed on the NHS, it must go through two different assessments. The first makes sure the treatment works and is safe. The second looks at whether it’s good value for money, so the NHS can make sure it’s using its resources wisely.
This was a crucial step forward, which we celebrated with the community. We spoke to Stephen, dad to eight-year-old Sam, who shared
“When givinostat was conditionally approved in December last year, we couldn’t believe there was a treatment Sam could receive that would hopefully slow the progression of his condition. Sam is still able to walk short lengths, so givinostat could keep him on his feet for longer – prolonging his independence would be the most amazing thing.”
Givinostat must now be assessed to check its good value for money. The process is different across the UK. In England, Wales and Northern Ireland, the National Institute for Health and Care Excellence (NICE) reviews the data, while in Scotland, it’s the Scottish Medicines Consortium (SMC). The pharmaceutical company behind givinostat, ITF Pharma Ltd (Italfarmaco Group in the UK), has already applied to NICE and confirmed they will be applying to the SMC in June. We don’t know exactly when NICE and SMC will make their decisions but we expect it to be later this year.
Both assessments are supposed to happen close together, so new treatments can get to the people who need them as quickly as possible. But in reality, that’s not always the case. Sometimes there are months, even years, between the first and second assessment – keeping treatments just out of reach.
Accessing treatments while assessments are completed
As the assessments are known to take a long time, many companies offer the treatment free of charge to the NHS, for eligible patients, through what’s called an Expanded Access Programme (EAP). However, since the treatment has not yet been approved by NICE or the SMC, the NHS doesn’t have to take part.
As a result, there’s no central system to help local NHS Trusts and Boards run the programme. There’s also no extra funding to cover the additional costs of giving the treatment safely or to provide more staffing to support this. This means access to potentially life-changing treatments through EAPs is often limited, inconsistent, and hard to come by.
Italfarmaco Group opened an EAP for givinostat in November 2024 – but so far, only a handful of people across the UK have been able to receive it.
Stephen and Sam are just one of many families struggling to access givinostat through the EAP.
“It’s almost six months since this was announced, and Sam is still waiting. Still getting weaker. We haven’t been given any idea of timeframes for when Sam might be able to receive the treatment. His consultant called us a couple of weeks ago and said that it’ll hopefully be available to Sam “soon”, but what if that’s not soon enough?”
Why is it so difficult to offer EAPs?
How complicated and expensive an EAP is really depends on the treatment. In every case, extra time from nurses and doctors is needed to check who’s eligible, prescribe the treatment properly, and keep an eye on any side effects. If the treatment has to be given in a special way or needs regular monitoring because of serious side effects, the costs can go up a lot. When that happens, only specialist centres are able to run the EAP, which means far fewer places can offer the treatment.
As there’s no central system for EAPs, it’s up to each local NHS Trust or Board to decide whether a centre can take part and to provide any extra support that’s needed. This means each centre has to apply individually, which can be a slow and complicated process. It also takes doctors and nurses away from caring for patients while they handle the paperwork.
Why is the givinostat EAP so difficult to offer?
Taking this into account, delivering givinostat through the EAP is difficult. Only the 24 specialist neuromuscular centres that form part of the NorthStar network are able to apply, and extra staff and resources are needed to make it happen. This includes:
Clinician time to apply for permission to run the EAP.
Nurse and clinician time to check if patients are eligible to take part, prescribe the treatment correctly and make any changes if patients experience side effects.
Givinostat can cause side effects in some people, so people taking givinostat must be closely monitored with regular blood tests.
Extra capacity to support regular blood tests.
Extra capacity in hospital pharmacies to ensure that the right dose is dispensed safely to each patient.
What are we doing to support access to givinostat?
Since the EAP opened in November 2024, we have been in discussions with NHS Trusts and Boards across the UK. From these conversations it’s clear that most centres still lack the resources needed to deliver the EAP. While planning discussions are ongoing, most centres have been unable to provide a timeline or clarity about when or how families would be reassured on any future access arrangements.
We’re stepping up our efforts and pushing for the timely, fair access families deserve. We share the frustration that many families are currently facing in trying to access givinostat and we recognise the emotional toll the uncertainty and delays to accessing new life-changing treatments can have.
We’re actively engaging with the UK Government, NHS Trusts and Boards and the media to ensure local neuromuscular teams have the resources needed to join the EAP and make sure all eligible boys receive givinostat as soon as possible. This includes reaching out to Chief Executives of NHS Trusts and Boards, Chief Medical Officers, Chief Pharmaceutical Officers, Health Ministers and supportive Members of Parliament across England, Wales, Scotland and Northern Ireland.
We’re also working in partnership with the charities Action Duchenne and Duchenne UK to make sure the community’s voice is heard during the NICE and SMC assessment of givinostat.
Tackling the problems in accessing treatments
“Every day we wait, more of Sam’s strength disappears. And once it’s gone, it doesn’t come back.”
This heart-wrenching reality is one that families like Sam’s are living every day, as they wait for treatments that could make a difference.
We’ve made advances that would have been unthinkable just 10 years ago – finally starting to discover potentially life-changing treatments for some muscle wasting and weakening conditions. And there are even more treatments on the horizon. But for these treatments to be effective, we must have the resources and capacity in the health system to give them as quickly as possible. This must be made a priority so that more people, like Sam, can access the treatments they need.
Take action
Engage with clinicians and NHS bodies
Contact your NHS Trust or Health Board: These are the decision makers – it is up to each local NHS Trust or Board to decide whether a centre will take part in the Expanded Access Programme. Ask if the treatment is under local review, available via a clinical trial or special funding pathway. To contact your local NHS Board or Trust, visit the official NHS website for your country (England, Scotland, Wales, or Northern Ireland), search by postcode or region, and use the contact details listed for your local provider. If you would like a template letter to send to your NHS Trust or Health Board, email info@musculardystrophyuk.org.
Talk to your clinician or specialist: They may be able to advocate on you and your child’s behalf.
Contact your parliamentarian
Write to or ask to meet with your MP/MSP/MLA: Ask them to raise your concerns in Parliament, with NHS decision-makers, or submit written questions. If you would like a template letter to send to your MSP, MLA, or MP, email info@musculardystrophyuk.org.
Contact your local press
Contact your local newspaper, TV and radio and share your story. Many families have done this already to raise awareness of the issue.
Connect with other families facing similar challenges
Peer networks can provide emotional support, share practical advice, and offer insights into navigating the system more effectively. We have Duchenne and parents and carers WhatsApp groups, and we also have a parents and carers Facebook group. Find out more.
Submit a formal complaint
You can file a formal complaint through the NHS complaints process. To make a complaint to your NHS Board or Trust, you can contact their Patient Advice and Liaison Service (PALS) or complaints department directly (usually by phone, email, or an online form). Details are usually available on their official website. You can escalate your complaint to the Parliamentary and Health Service Ombudsman if the situation remains unresolved.
We understand this is a difficult time for families trying to access givinostat. If you need support please do reach out to our helpline, call 0800 652 6352 or email info@musculardystrophyuk.org.
