Families urge HSE to make life-changing drug Givinostat available as soon as possible 

Parents warned the longer it takes for Givinostat to be made available, the higher the chance patients will become ineligible

Jennifer Cullen and Andrew Levin (Tallaght) who’s son AJ will now be able to avail of the drug. Picture: Sam Boal/Collins Photos

Duchenne muscular dystrophy families have said they will not celebrate a drug to treat the disease being recommended for approval until it is “in the fridge”.

The HSE Drug Group has said Givinostat should be recommended for reimbursement to treat the rare disease, a severe, progressive genetic disorder that weakens the muscles.

However, families of children with Duchenne muscular dystrophy (DMD) have now urged the HSE senior leadership team to rubber-stamp the drug without delay, warning that children have already become ineligible in the year since their campaign started.

In a briefing for TDs and senators, parents were cautiously optimistic following the Givinostat recommendation but begged for the drug to be made available within weeks.

Karen Thompson, whose two sons Conor (10) and Dean (9) both have DMD, said the news is a “bittersweet celebration” as she questioned why families should have to fight this hard to get treatment for their children.

She said she hoped that Givinostat would be available to the 100 boys who have DMD before the end of the summer.

“That's when we celebrate; When we have it in our fridges,” Ms Thompson said.

The families are imploring the HSE to follow the “NICE” guidelines used in the UK, which dictate that children are eligible for the drug if they can walk or stand, either supported or unsupported, rather than limiting it to only children who can walk.

Michael Brogan, dad of DMD patient Fionn, aged 10, warned the longer it takes for Givinostat to be made available, the higher the chance patients will become ineligible.

“Unfortunately, because Ireland doesn't have an early access programme like so many other countries, there are boys that have now missed the window [for Givinostat],” he said.

“[That] is really, really sad, and it's quite unforgivable. 

Michael and Naive Brogan (Dublin) who’s son Fionn will now be able to avail of the drug.  Picture: Sam Boal/Collins

"There's an opportunity now, after the good news [from the HSE] to really expedite that and finish this process, so our boys receive this drug.

“There is a real risk over the next coming weeks and the next couple of months that other boys could lose out. The responsibility is now back with the HSE and the decision makers to roll it out as quickly as humanly possible.”

Fianna Fáil Senator Teresa Costello, who has been campaigning for the Givinostat for the last year, said it has been “really difficult”. She said she would like to see the drug delivered within six weeks.

In the Dáil, her party colleague Pádraig O’Sullivan said that while this was good news, there are other drugs for rare diseases, including Skyclarys for Friedreich ataxia, which are needed for people without access to treatment.

He expressed concern that it is taking an average of 800 days for rare disease drugs to be approved, while other countries in Europe approve them twice or three times as quickly.

“We are consistently laggards in the league table when it comes to reimbursing these drugs,” Mr O’Sullivan said.

“There must be dozens and dozens of parents at home and families at home who are looking on, obviously, while they wait for their drug to be reimbursed or to be assessed. It's a cruel, horrible situation for any family to be. It's inhumane.”

• Louise Burne is Political Correspondent