- NICE recommends Duvyzat for Duchenne muscular dystrophy on the NHS.
- The drug is expected to help children with DMD lead an independent life for longer.
- NICE welcomed the treatment but opined that more long-term evidence is needed.
The National Institute for Health and Care Excellence (NICE) has approved givinostat (also named Duvyzat and manufactured by ITF Pharma) for the treatment of Duchenne muscular dystrophy (DMD), a chronic, progressive and a fatal genetic disorder which mainly affects boys.
Around 530 people in England will benefit from givinostat following the successful negotiation of a commercial deal to make the medicine available on the NHS.
DMD is caused due to the lack of protein dystrophin, which leads to muscle degeneration. The symptoms generally appear from the age of three.
For patients, this leads to disruption of free movements, leaving them dependent to carers. In worst case scenarios, the disease grows to affect the heart and lungs making life support machines a necessity.
Givinostat, the third DMD treatment suggested by NICE, can be used to treat DMD from the age of 6 years for patients who has difficulty in walking or standing with or without support.
It will slow the progression of the disorder despite the type of DMD.
The medicine available since November 2024, will be immediately funded via the Innovative Medicines Fund.
Givinostat was suggested after successful clinical trials done on populations, which proved that it increased the possibility of DMD patients to move freely by 5 years on an average unlike other treatments such as corticosteroids and supportive care.
“Givinostat is a promising treatment, so we are delighted the company and NHS England have been able to reach a deal that recognises its potential but also the uncertainties that remain around the extent of the benefit it provides,” commented the director of medicines evaluation at NICE, Helen Knight.
"This is exactly what our NHS is here to do: secure the best available treatments for patients and ensure they are available quickly and fairly,” added Health Innovation and Safety minister, Dr Zubir Ahmed.
However, NICE stated that more studies need to be done on the medication as there are various challenges around it, such as the difficulty in fully understanding the effectives of givinostat on patients’ quality of life as well as the carers.
