Givinostat – why is accessing new treatments so difficult?

25 February 2025

It’s an exciting time for research into muscle wasting and weakening conditions. Researchers are starting to find treatments which could slow disease progression. But getting these treatments into people who need them is a long, complicated and often difficult process. The complex process is currently making it difficult for people with Duchenne muscular dystrophy to access a new treatment, called givinostat. Improvements are needed so that everyone can access the treatment they need, when they need it.

Givinostat, also known as Duvyzat, was conditionally approved by the Medicines and Healthcare products Regulatory Agency (MHRA) in the UK in December 2024. However, it’s not yet available for routine use on the NHS.

For this to happen, it must be recommended by the National Institute for Health and Care Excellence (NICE) in England, Wales and Northern Ireland; and the Scottish Medicines Consortium (SMC) in Scotland. NICE and the SMC review how effective the treatment is and weigh it against the cost of the treatment. The process has started for givinostat, but a decision won’t be made until later in 2025.

Accessing treatments while decisions are made

People with muscle wasting conditions, like Duchenne muscular dystrophy (DMD), don’t have time to waste. Accessing treatments as early as possible could make a significant difference in quality of life. Programmes, such as Expanded Access Programmes (EAP), can help people get free-of-charge access to treatments while the NICE and SMC process continues.

An EAP for givinostat is open in the UK for specific eligible patients with DMD. However, we are aware that people are struggling to access the treatment through the EAP.

Difficulties with expanded access programmes

In these programmes, the manufacturer provides the treatment free-of-charge. However, the local NHS Trust is responsible for covering the costs associated with administering the treatment or monitoring patients while they receive the treatment.

To administer givinostat safely additional staff and resources are needed:

• Givinostat can cause some side effects in some people, so people taking givinostat must be closely monitored with regular blood tests.
• Additional nurse and clinician time is needed to check if patients are eligible to take part in the EAP, prescribe givinostat correctly, monitor blood test results and make any changes if patients experience side effects.
• Extra capacity will be needed to support regular blood tests.
• Extra capacity will also be needed in hospital pharmacies to ensure that the right dose is dispensed safely to each patient.

As a treatment in an EAP is not yet recommended by NICE or SMC, local NHS Trusts are often unable to provide the additional resources needed to administer and monitor patients receiving the treatment.

There also isn’t a centralised system in place for EAPs. Each neuromuscular centre must apply to their local NHS Trust for permission to take part in the EAP. This application process can be lengthy, taking valuable time away from clinicians.

Finding the additional resources

Clinicians around the UK are working tirelessly to find a solution. Conversations with local NHS Trusts to get permission to take part and see if the additional resource needed can be provided are ongoing. Without permission and additional resource, neuromuscular centres cannot participate in the EAP, delaying access to givinostat.

What we are doing

We want all people in the UK with muscle wasting and weakening conditions to have access to the best treatment. As quickly as possible. As part of our Access to Treatments campaign, we work with our community, healthcare professionals, researchers, other charities and patient groups to make sure the views and experiences of people with muscle wasting conditions are heard.

Getting treatments to the people who need them is a complex process. We feel it’s important to support the clinical community as they navigate this process to help provide access to treatments in a safe and sustainable way. We have spoken with members of the NorthStar Network to better understand the challenges that they are facing.

We’re also writing to key leaders in the NHS across the England, Wales, Scotland and Northern Ireland about the measures being taken to support the rollout of the EAP and ensure equitable access across different regions and NHS Trusts. We’re also asking what plans they have in place to ensure there will be a smooth rollout to patients in a timely manner if approved by NICE and SMC.

As research continues to move forward, more treatments will hopefully become available for many more muscle wasting and weakening conditions. We will continue to support and amplify the voices of people with muscle wasting conditions in the assessment of new treatments, as well as work to ensure the system has the capacity to administer them.