Access to treatments

London Assembly unanimously backs Duchenne UK’s Time is Muscle campaign 

On Thursday 5 June the London Assembly voted unanimously for a motion calling for a new medicine to be rolled out to all patients suffering from a cruel muscle-wasting disease.

The medicine, givinostat, is currently available for free to the NHS, and is proven to slow the progress of Duchenne muscular dystrophy (DMD) in clinical trials. Only patients who can walk are eligible for the free treatment, but families and campaigners are fighting for access for both ambulant and non-ambulant people with DMD. 

The motion, proposed by James Small-Edwards AM, London Assembly Member for West Central, calls on the Mayor to liaise with NHS England, the Department of Health and Social Care, and London’s NHS Trusts to ensure resources are put in place so all eligible DMD patients in the capital can benefit from access to givinostat.  

Currently, no DMD patient either ambulant or non-ambulant in London has accessed the medicine through the Early Access Programme (EAP) – which provides givinostat for free to the NHS – but it is already available to people with DMD through the NHS in other parts of the UK such as Scotland, South Wales and Leicester. 

The motion also recognised the tireless work of sister charities Duchenne UK and Joining Jack in campaigning for access to givinostat for their community. 

Givinostat is a new medicine for DMD which was approved in December 2024 by the Medicines and Healthcare products Regulatory Agency (MHRA) for everyone with DMD aged 6 and over, and is being made available for free to the NHS by the manufacturer, ITF Pharma UK, through a type of compassionate use scheme they call the EAP. The scheme allows specialist neuromuscular clinicians to access givinostat for their patients without the NHS having to pay for the medicine – until NICE makes a decision on its cost effectiveness. 

The EAP is currently open to ambulant patients, but the MHRA market authorisation approves givinostat for everyone with DMD aged 6 and over, and the DMD community is campaigning for non-ambulant boys who could benefit from givinostat to have access too. 

Givinostat is an ‘HDAC inhibitor’. This means that 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. 

James Small-Edwards AM, said;

“Everyone living with Duchenne muscular dystrophy deserves timely access to the most promising treatments available. The givinostat Early Access Programme offers real hope for Duchenne families but it’s currently a postcode lottery. We’re calling on the Mayor and NHS partners to make sure ambulant and non-ambulant patients in London are not left behind.”

Emily Reuben OBE, the CEO and Founder of Duchenne UK, said;

“Time is muscle, and every day access to givinostat is delayed means another patient who will no longer be eligible for this proven medicine. We are thankful to James and his fellow Assembly Members for supporting our Time is Muscle campaign, and hope their efforts will quickly accelerate access to givinostat in London for ambulant and non-ambulant patients. ”

Duchenne UK has been campaigning for access to givinostat with their ‘Time is Muscle’ campaign since they first learned it was being provided for free through the EAP. You can learn more about their campaign here.