Parents plead for approval of 'vital drug' to slow down their son's muscular dystrophy

Archie Ennis: His parents have so far raised nearly €600,000 to fund specialist gene therapy.

The mother of a child seeking treatment abroad for an incurable muscle-wasting disease is urging the Government to help secure a vital drug which would slow down the progression of her son's disease.

Earlier this year, Una Ennis took to social media with a heartfelt plea for funds so she could access specialist treatment abroad, following her seven-year-old son Archie’s muscular dystrophy diagnosis.

The rare condition currently affects 110 boys in Ireland, with no cure.

However, specialist gene therapy is available in other countries, such as the US, to slow the disease's progression, with costs for such treatment starting at €3m.

Ms Ennis and her husband Kenneth have so far raised nearly €600,000 in the hope of securing Archie such treatment but are calling for medication to be made available here, specifically a drug called givinostat
which improves muscle repair.

Ms Ennis told the Irish Examiner: “We are still fundraising for the gene therapy, and we are chasing the Government and ministers to help put the drug givinostat in place.

“We have been dealing with politicians who are working with the European Medicines Agency (EMA).

“Hopefully we can get it approved, we will know more in June, but obviously then, the pharmaceutical companies want the Government to fund it, so it is a long process.

I have been told that timing is of the essence though, the sooner Archie goes on something like this the better chance he has of survival.

“His muscles are weak, and he fell recently so he has a limp, it’s just going to be the way it is”.

Givinostat is a drug that blocks the activity of histone deacetylase, to help improve muscle repair.

It works by reducing inflammation and the build-up of scar tissues and ultimately slows progression.

The liquid medication is not on the market in Ireland but is available in some EU countries, as well as some parts of the UK.

Recently, a 12-year-old boy called Alfie Pentony from Newry, Co Down, spoke on BBC radio about how he could not use givinostat because even though it is available in other parts of the UK, it is not available in the North.

Alfie thus has to travel to Newcastle-upon-Tyne for treatment, and also takes steroid medication to slow down muscle weakness.

His father, Jamie Pentony, said givinostat could slow his son's incurable condition down, but the Belfast Trust did "not have the capacity" to offer it.

Ms Ennis said: “It isn’t fair that these children can’t access these vital drugs. We are meeting the EMA in June and if we can get it approved then at least that is a start”.

In a statement the HSE said “To date, neither the EMA nor the HPRA have granted marketing authorisation for givinostat for any indication. A national assessment and decision process cannot commence in the absence of a marketing authorisation.

“The HSE must comply with the relevant legislation when considering investment decisions around medicines.”

In response, the HPRA said: "At this time, Giviniostat has not been authorised by the EMA or the HPRA."

Donations for Archie's treatment can be made [https://www.gofundme.com/f/4s5t9y-please-help-our-boy] here.