PKU drug to be made available to Irish patients at third time of asking

HSE has agreed to reimburse Kuvan for up to 20pc of patients with rare metabolic disorder

Families of people with phenylketonuria (PKU) have expressed their delight after the Health Service Executive agreed to fund the only drug treatment for the rare metabolic disorder for some patients.

From next week, Kuvan will be made available on an individual basis through medical specialists for a minority - 10-20 per cent - of Irish patients with PKU.

The drug was rejected on cost-efficiency grounds in 2009 and 2017 but has made it through the drug approval process this year after the HSE leadership team approved it for reimbursement in the Irish health service.

It received EU regulatory approval in 2008 and has since been widely used across Europe. In Ireland, it is being made available for specific indications according to an agreed protocol developed between doctors and patient representatives.

Ireland has one of the highest rates of PKU which requires sufferers to adhere to a strict low-protein diet in order to avoid irreversible mental and physical disabilities.

Over 700 patients with PKU are receiving hospital care in Ireland. The National Centre for Pharmacoeconomics had previously estimated the five-year cost to supply the drug at €12.66 million.

Fergus Woodcock, chairman of the PKU Association of Ireland said patients were delighted the treatment was now available here and felt empowered from their involvement with the approval process.

Noting Friday is International PKU Day, Minister for Health Simon Harris said the HSE decision would come as a "huge relief" for families affected by the disease.

A PKU diet removes foods such as meat, fish, poultry, dairy products, bread, flour, rice, pasta, yoghurt and chocolate. In addition, all other foods consumed must be strictly monitored for protein levels.

The prevalence of PKU in Ireland is one in 4,500 babies, twice the average in the rest of Europe. Ireland was one of the first countries to introduce the heel prick test at birth for the disorder, almost 50 years ago.