Landmark approval of new treatment for DMD in Scotland
Duchenne UK is proud to welcome a major breakthrough for families affected by Duchenne muscular dystrophy (DMD) in Scotland, as the Scottish Medicines Consortium (SMC) approves a new treatment for use on the NHS in Scotland.
In guidance published today (8 December), the SMC has accepted givinostat for the treatment of children aged six and older who are still able to walk. This marks a pivotal moment for people living with DMD—a rare, devastating genetic condition that causes progressive muscle weakness and eventually impacts the heart and lungs.
A victory for the community
Duchenne UK played a key role in the SMC’s assessment of givinostat and in helping secure this breakthrough. We worked alongside Muscular Dystrophy UK and Action Duchenne on a joint submission to the SMC. As designated patient groups, we worked together to represent the community throughout the assessment process, as well as nominating two parents of children and young people living with DMD to provide their views on the treatment and share their lived experience.
Duchenne UK’s nominated patient expert, Mhairi Harvey, mother to a son living with DMD, spoke powerfully about the urgent need for this treatment and the difference it could make to families during the SMC’s Patient and Clinician Engagement (PACE) meeting.
Our Project HERCULES built critical evidence on the real-world impact of DMD, which was used in our submission to the SMC to help demonstrate the potential quality of life benefits of the medicine. Fleur Chandler, who chairs Project HERCULES, also represented Duchenne UK at the SMC Committee meeting during which she was able to draw on her dual experience as a parent caregiver to her son living with DMD, and her professional background as a health economist, to make a compelling case for the approval of givinostat.
In the early stages of the drug’s development, our DMD Hub also hosted givinostat trials.
Emily Reuben OBE, Co-founder and Chief Executive of Duchenne UK, said:
“This approval represents a significant breakthrough for people living with Duchenne muscular dystrophy and their families in Scotland.
“Driving medical research and accelerating access to new treatments is at the heart of our mission to one day end Duchenne. Today, we see that work delivering real progress for patients. In the last year, we’ve gone from having extremely limited treatment options available to now having two approved medicines, offering hope to hundreds of families, and in the case of givinostat, the promise of slowing down the progression of this devastating disease.
“Givinostat must now also be urgently approved for use in NHS England by NICE and made available to all eligible patients across the UK, to ensure that everyone who could benefit from this medicine is able to do so without further delay. We also continue to explore how patients who’ve already lost their ability to walk can be given the opportunity to potentially benefit from this new treatment.”
What does this mean for patients in Scotland?
The SMC’s decision grants approval for ‘restricted use’ of givinostat in Scotland, as it is limited to patients who can walk when they first start treatment. This includes patients who become unable to walk whilst receiving the drug, who can continue to be treated with it.
We continue to explore how patients who’ve already lost their ability to walk can be given the opportunity to potentially benefit from this new treatment and are in discussions with ITF Pharma UK, clinicians and regulators on this.
What does this mean for patients in other areas of the UK?
The SMC is an independent body that makes decisions about whether new medicines can be prescribed routinely in Scotland. Today’s decision will therefore not change the availability of givinostat for patients under the care of the NHS in England, Wales, and Northern Ireland.
Givinostat remains under review by the National Institute for Health and Care Excellence (NICE) for use in NHS England and guidance is yet to be issued. Once NICE issues guidance, it is generally applied in Wales and Northern Ireland, as well as England. As NICE and SMC decisions are unrelated it is not uncommon for one body to make a decision on whether to approve a medicine for use, before the other. They can also make different decisions.
We continue to press NICE for an update on its ongoing appraisal of givinostat, urging them to bring it to a swift conclusion for the sake of families still waiting and hoping for positive news.
We will keep the community informed and provide any updates from NICE as soon as we are able to.