Teresa Costello (Fianna Fail)

I want to be clear I am no expert on Duchenne muscular dystrophy or DMD as it is commonly referred to. However, since a young boy from Tallaght was diagnosed with this condition, I have come to understand the immense challenges faced not only by the patients but by their families as well. From what I gather - and I am open to correction on this - treatment options in Ireland are limited to not much more than steroids and a wheelchair.

Duchenne muscular dystrophy is a progressive neuromuscular disease that causes the weakening of muscles over time. It affects almost exclusively boys, with symptoms typically appearing between the ages of one and three. In Ireland today, around 110 people are known to be living with DMD. Most of them are under the age of 18. In the UK, approximately 100 boys are born with the condition each year. For a child living with DMD, issues, they say, can include struggling to walk, run or jump like their peers, difficulty standing up, delayed speech, needing help to climb stairs and sometimes facing behavioural or learning challenges. As the disease progresses, most children will need a wheelchair by the time they are 12. Some will develop scoliosis. By their mid-teens, many experience heart complications and, by their late teens or early 20s, breathing problems often emerge, making the condition life-threatening.

There is, however, a glimmer of hope in the form of a new drug called givinostat. It is marketed as Duvyzat and it offers a breakthrough. It has been shown to slow the progression of muscular degeneration in those with DMD. Clinical trials have met both primary and secondary endpoints, demonstrating its effectiveness. In December 2024, the UK's Medicines and Healthcare Products Regulatory Agency approved givinostat for use in boys aged six and over. It is currently being provided through the expanded access programme at no cost to patients via local NHS trusts.

This drug, however, is not yet in Ireland. Givinostat should have its full EU licence by approximately the end of June. The next step would be that the manufacturer will make a submission for a higher technical assessment with the National Centre for Pharmacoeconomics, NCPE. In Scotland, which is not very far away, I have been told - and I am open to correction on this - that, as regards the decision-maker similar to the NCPE in Ireland, the turnaround time from submission to availability is about five months. I would like to push for a similar turnaround time given the impact time has on the eligibility of patients for this treatment. If you are looking at your child who has been diagnosed with this, you are looking at a clock. Givinostat does not cure DMD; it slows its progression. It cannot turn back the clock or undo the damage done but it can slow it down, giving these boys more time on their feet without the need of a wheelchair.

There is a race against time for these children. I read a press release a few days ago, on 25 April, confirming that the European Medicines Agency, EMA, has recommended a conditional marketing authorisation for Duvyzat across the EU. I would like to know the status on getting givinostat to our Irish patients.