Monday 15th January, 2018  

HSE DECISION ON ONLY DRUG TO TREAT PKU THIS THURSDAY

PKUAI HOPES FOR POSITIVE OUTCOME FROM PROTRACTED PROCESS

Kuvan has been widely available throughout Europe since 2009, but is still not available here in Ireland. 

PKUAI is greatly encouraged that best practice for PKU is in sight and continue to urge the HSE to take advantage of this unique opportunity.  Approval for the ‘small group’ of Irish PKU patients who can benefit greatly from the drug, at a relatively small cost, will result in a huge increase in quality of life. 

The PKUAI acknowledge Kuvan will not work for all PKU people due to the complex nature of the disease and predominant ‘Celtic’ PKU gene types here in Ireland, referred to as ‘classical’.  

Fergus Woodcock, Chairman, PKUAI commented “We hope the HSE seize this opportunity and delivers a swift conclusion to what has been an excessively protracted process for the PKU community.  A positive outcome will transform the lives of some members of our community – which will be a hugely successful outcome.  The PKUAI will support metabolic clinicians (adult and child) to identify those who are most likely to derive the optimal clinical benefit”.  

Fergus added: “Whatever the outcome, PKUAI will continue to educate and raise awareness for PKU, seeking best practice and best care for those with the disease.  We recognise the acute need for a new medicines review to be established. PKUAI hopes that, as part of the new system, meaningful patient perspectives are gathered and formally factored in when approving new and innovative drugs now and going forward, and the process is accountable and transparent so all rare disease groups will benefit”.

PKUAI hopes this week will bring a positive outcome for the PKU community and a positive start to 2018.

ENDS.

For further information contact: Emer O’Reilly @ +353 86 859 3658 emer@orpr.ie 

For further information on PKU check out PKUAI web-site