A significant milestone for the Duchenne community in Scotland as a new treatment option due to become available on the NHS.
We are delighted to share the positive news that the Scottish Medicines Consortium (SMC) has approved the medicine givinostat for use within NHS Scotland. This decision marks a vital step forward in ensuring families living with Duchenne muscular dystrophy (DMD) have access to treatments that have the potential to slow the progression of muscle degeneration.
For our community, every new treatment option offers hope, and Action Duchenne welcomes this decision to expand the therapeutic landscape for families in Scotland.
Who is eligible for this treatment?
The SMC has accepted givinostat for restricted use. It is important for families to understand exactly what the designation of ‘restricted use’ means for eligibility.
According to the guidance issued, givinostat is approved for patients who meet the following criteria:
- Age: Patients must be 6 years old or older.
- Ambulatory Status: Patients must be able to walk at the time they first start the treatment. This includes patients that start treatment while able to walk but later become non-ambulant.
Important Note on Continued Use: The SMC has clarified that while a patient must be ambulatory to start the drug, they are permitted to continue taking it even if they later lose the ability to walk. This ensures that patients can continue to benefit from the treatment as their condition changes.
The to the Decision Explained public information summary can be found here.
What does the SMC decision mean for you?
This approval means that Givinostat is now due to be made available for prescribing on the NHS in Scotland.
The treatment was approved by the Medicines and Healthcare products Regulatory Agency (MHRA) in December 2024 and remains under review by National Institute for Health and Care Excellence (NICE) for use in NHS England.
We understand that navigating treatment options can be complex. If you wish to discuss eligibility, your next step will be to contact your neuromuscular clinician to discuss if it is the right treatment for you or your child.
A victory for the community
Charities Duchenne UK, Muscular Dystrophy UK, and Action Duchenne provided a joint patient group submission to the SMC in support of the treatment’s approval. As designated patient experts, the three organisations worked together to represent the community throughout the assessment process, alongside parents of children and young people living with DMD, who bravely shared their lived experience and highlighted the potential benefits and importance of this new treatment.
A note from our Team
We know that Duchenne is a relentless condition, and the search for effective treatments is a journey we walk together. While we celebrate this approval, we remain acutely aware of those in our community who may not meet these specific criteria. Such news can be difficult to receive and if you feel you need to talk or require support, please reach out to us at support@actionduchenne.org.
