Givinostat, a treatment that slows Duchenne muscular dystrophy progression, to be accessible through NHS Scotland for eligible boys and young men.
The Scottish Medicines Consortium (SMC) has accepted givinostat (also known as Duvyzat) for restricted NHS use. This means the treatment will be available to boys and young men who are six years and over and can walk when they start treatment. Those who lose the ability to walk while receiving givinostat can continue treatment.
Givinostat is not a cure for DMD, but it has the potential to slow progression, helping families hold on to more of the everyday moments that matter and improving quality of life.
This marks another major milestone for families affected by DMD in Scotland. Until recently, corticosteroids were the only treatment option for most people with DMD. In the past year, two new treatments have been approved in Scotland – giving families more hope and choice.
“This is an incredible moment for families living with Duchenne muscular dystrophy in Scotland. After almost a year of working for access, seeing this treatment accepted for use in Scotland means so much.”
“This is a landmark moment for families in Scotland. People with muscle wasting conditions, like Duchenne muscular dystrophy, don’t have time to waste. Accessing treatments as early as possible can make a significant difference and with givinostat now accepted for NHS use, families gain access to a treatment that may slow progression and help maintain quality of life. To see two treatments accepted in Scotland within a year is extraordinary progress for the DMD community.”
This decision only applies to people with DMD living in Scotland. In England, givinostat is still under review by the National Institute for Health and Care Excellence (NICE), and Wales and Northern Ireland usually follow NICE decisions. On 16 December, NICE announced they had paused the assessment to allow the company that makes givinostat and NHS England to have discussions about pricing.
We know families across England, Wales and Northern Ireland are eagerly waiting for news. As soon as we have more information, we will share with the community.
“Our focus now is ensuring families across the rest of the UK can benefit too.”
Givinostat is designed to help slow the progression of Duchenne muscular dystrophy (DMD). It blocks the activity of histone deacetylase – a molecule that is overactive in DMD and interferes with muscle repair.
By blocking this molecule, givinostat aims to improve muscle repair, reduce inflammation and scar tissue build-up, and ultimately help maintain muscle function for longer.
This achievement is the result of working together as a community. Throughout the assessment process, we partnered closely with charities, Duchenne UK and Action Duchenne, alongside families affected by DMD to make the strongest possible case for givinostat.
Together, we:
We also appreciate the constructive engagement from NHS Scotland and the Scottish Government throughout the process.
“We want to thank everyone who shared their powerful stories to show the real impact of this condition and the difference this treatment could make. Your voices were vital in securing access in Scotland.”
We’ll continue to work in partnership with the community as we focus on ensuring access across the rest of the UK.
