Givinostat can be used as an option to treat Duchenne muscular dystrophy in people 6 years and over who are ambulant (able to walk or stand, with or without support) at the start of treatment. Givinostat can only be used if the company provides it according to the commercial arrangement.
This recommendation is not intended to affect treatment with givinostat that was started in the NHS before this guidance was published. People having treatment outside this recommendation may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS healthcare professional consider it appropriate to stop. For children or young people, this decision should be made jointly by the healthcare professional, the child or young person, and their parents or carers.
Givinostat must be funded in the NHS in England for the condition and population in the recommendations, if it is considered the most suitable treatment option. Givinostat must be funded in England within 90 days of final publication of this guidance.
There is enough evidence to suggest that givinostat provides benefits and value for money, so it can be used routinely across the NHS in this population.
NICE has produced tools and resources to support the implementation of this guidance.
Duchenne muscular dystrophy is a rare and fatal genetic condition that starts in childhood. It causes muscles throughout the body to gradually stop working, leading to increasing disability over time and early death.
There is currently no cure for Duchenne muscular dystrophy. The usual treatment is best supportive care focusing on preventing and managing complications.
For this evaluation, the company asked for givinostat to be considered only for people who are ambulant (able to walk or stand, with or without support) at the start of treatment, to reflect the populations in the clinical trials. This does not include everyone who givinostat is licensed for.
Clinical trial evidence shows that givinostat slows down the loss of ability to climb stairs compared with corticosteroids alone. But, there is no evidence yet on whether givinostat helps delay breathing problems because the trial is still ongoing.
When taking into account the condition's severity, and its effect on quality and length of life, the cost-effectiveness estimates are within the range that NICE considers an acceptable use of NHS resources. So, givinostat can be used.