NICE has approved givinostat for some people with Duchenne muscular dystrophy in England after a commercial deal was reached to make the medicine available on the NHS. The decision means around 530 people are expected to benefit from access to the treatment, which is now being funded immediately through the Innovative Medicines Fund.
A new option for a severe and progressive condition
The treatment, also known as Duvyzat and made by ITF Pharma, can be used in people aged 6 and over who are able to walk or stand, with or without support, at the start of treatment. NICE said this reflects the populations studied in clinical trials rather than the full licensed population.
Duchenne muscular dystrophy is a rare, severe and progressive condition caused by the lack of dystrophin, a protein important for muscle cells. According to NICE, symptoms usually appear at around age 3 and the disease gradually leads to loss of walking ability, then sitting, and eventually greater dependence on carers and breathing support.
Evidence suggests walking ability may be extended
NICE said evidence from clinical trials suggests givinostat may increase the length of time people with Duchenne muscular dystrophy can walk by around five years on average compared with established clinical management, which includes corticosteroids and supportive care. The organisation also said uncertainty remains because of limitations in the available evidence.
Helen Knight, director of medicines evaluation at NICE, said the deal recognises both the medicine’s potential and the remaining uncertainties around the benefit it provides. Health Innovation and Safety Minister Dr Zubir Ahmed said the approval gives patients the power to slow the progression of the disease and thanked families who shared their experiences during the process.
The recommendation makes givinostat the third treatment NICE has recommended for the condition. NICE said the committee concluded the medicine would be a cost-effective use of NHS resources after applying the maximum severity weighting and taking into account the evidence available. For families affected by Duchenne muscular dystrophy, the decision offers another treatment option in a disease where there has long been limited choice.
Source: NICE news release
