Access to treatments

Change of mind at GOSH as they begin dosing givinostat through the Early Access Programme

Great Ormond Street Hospital, the UK’s largest paediatric hospital, has agreed to participate in the Early Access Programme (EAP) for givinostat, and to facilitate access for eligible patients immediately.

Last month the hospital said they would be unable to take part in the EAP due to capacity issues.

However, following a meeting in May between Duchenne UK’s CEO, Emily Reuben OBE, and the CEO of GOSH, Matthew Shaw and his team, the hospital has now agreed to take part.

GOSH have confirmed that they are able to offer givinostat to eligible patients, and that their clinical team will be contacting all patients who may be eligible for the treatment to let them know it will be discussed in their next appointment.

They aim to see all potentially eligible patients within the next four months, and are prioritising those patients who require the treatment most urgently or are at risk of losing ambulation. You can read more about GOSH’s announcement here.

Emily Reuben OBE, CEO and founder of Duchenne UK, said,

“I am very grateful to GOSH for agreeing to find the resources required to take part in the EAP. It’s plain to see that the demands on the NHS are huge, but by not neglecting a community with a rare and severe disease like Duchenne muscular dystrophy, GOSH is demonstrating that the most vulnerable don’t need to be hit the hardest.

“I applaud the team at GOSH for making this happen. Time is muscle, and our boys cannot afford to wait for access to this treatment.

“We are continuing our passionate campaign to get the EAP rolled out everywhere.”

Givinostat is not a cure, but it has been proven to slow the progression of DMD in clinical trials. Givinostat was granted market authorisation in December 2024 for everyone with DMD aged 6 and over, and is currently being assessed by NICE for cost-effectiveness before it can be made available through the NHS. While it is being assessed by NICE, the company behind givinostat – ITF Pharma UK – are providing the medicine to the NHS for free through the EAP.

To take part in the EAP clinics must screen patients, take blood tests for monitoring, and adjust the dose if necessary. This, along with other burdens on clinic and pharmacy resources has prevented the full and fast roll out of the EAP.

Duchenne UK has been advocating for participation in the EAP through their Time is Muscle campaign since they first learned about the programme at the end of 2024. To be eligible to take part in the EAP, patients must be ambulant. Every day that access to givinostat is delayed means more patients who could have been eligible losing out on access.

To learn more visit www.duchenneuk.org/timeismusclecampaign.