Time is muscle
Join our fast-paced and passionate community driven campaign, to give our patients access to a new treatment givinostat
Join our fast-paced and passionate community driven campaign, to give our patients access to a new treatment givinostat
Duchenne muscular dystrophy (DMD) is a cruel muscle-wasting disease. But there is hope.
A new medicine for DMD has been approved in the UK. It is not a cure, but it could slow the progression of DMD. It’s called givinostat, also known by the brand name Duvyzat, and has been here for several months.
But many people with DMD can’t access it. While it has been approved by the Medicines and Healthcare products Regulatory Agency (MHRA), it won’t be available routinely on the NHS until the National Institute for Health and Care Excellence (NICE) decide whether they want to pay for it.
But eligible patients could access it now! Givinostat is available for free to the NHS through an Early Access Programme (EAP) until NICE reaches a decision.
We’re campaigning to fix that. We want access to givinostat for everyone eligible in the UK with DMD so they all have the chance to keep their muscles moving for longer.
It’s not because their neuromuscular consultants think some patients couldn’t benefit. They do.
It’s not because of the cost. It’s currently free to the NHS through the EAP.
Many people with DMD aren’t being given givinostat for two reasons.
(1) For those that are eligible through the EAP, many clinics say they don’t have the admin capacity to process the paperwork
(2) The eligibility criteria for the EAP, means that non-ambulant (not walking) people with DMD can’t be prescribed it
We held a webinar in December 2024 to discuss the issues facing Trusts in providing access to givinostat via the EAP
We have two asks:
(1) For Trusts to give their neuromuscular teams administrative support so they can prescribe givinostat to patients who are eligible for it through the EAP.
(2) The MHRA gave approval on 20 December 2024 to givinostat for patients aged six years and older who are ambulatory (walking) and conditional approval for people who are no longer ambulatory. We are now calling for it to be approved for use on the NHS for people with DMD who are ambulant and non-ambulant.
Our campaign only started in November, but we have made so much progress thanks to our amazing community
We held an event in parliament on 22 January, which 25 MPs attended including Andrew George MP, who is a member of the House of Commons Health and Social Care Committee, who all said that they would support our campaign. We had already been in contact with MPs, and Aphra Brandreth MP has sent a question to the Department for Health and Social Care asking what steps it was taking to support specialist neuromuscular clinics that wish to take part in the givinostat Early Access Programme, as a result of Jill and Richard Binns, whose son Jack has DMD, contacting them. While Freddie van Mierlo MP sent a question to the Secretary of State for Health and Social Care asking how many patients were receiving givinostat on 10 December, and what would he do to remove barriers to accessing it. This was the result of Alex Clarke, whose son Ben has DMD, contacting him.
Eli Crossley, Jack Johnson and Benjamin James, three young men with DMD, sat face to face with the Secretary of State for Health and Social Care, the Rt Hon Wes Streeting MP, for a full 45 minutes to discuss with him the challenges the DMD community is facing right now at our parliamentary event for MPs. He said that he would help us gain urgent access to givinostat. They also spoke to another cabinet member at the event, Rt Hon Lisa Nandy MP, Secretary of State for Media, Culture and Sport, about our #TimeisMuscle campaign.
We’ve written to all 24 NHS Trusts which are part of the NorthStar clinical network, in collaboration with MDUK, urging them to give clinics the resources they need to take part in the EAP.
An incredible 200 of you have written to the MHRA – which is currently reviewing givinostat. These are incredible numbers for a rare disease, and we know that decision makers are taking notice.
After coming to our event for MPs, Clive Efford MP, whose constituent has DMD, asks question in House of Commons about access to givinostat
We have already made important progress, we we need your help to keep up the momentum and pressure for access to givinostat!