Drug makers 'disappointed' over rejection

Dónal Nolan

The company behind the only drug available to treat the serious life-limiting condition PKU says it was 'disappointed' by the State's decision not to fund the drug.

In a statement to The Kerryman, company Biomarin - the manufacturer of drug Kuvan - said that it is looking forward to discussing the treatment with the HSE, however. The statement comes little over a month after the National Centre for Pharmacoeconomics (NCPE) dashed affected families' hopes that the State would fund the drug Kuvan.

At least two Kerry families affected by the rare genetic condition Phenylketonuria (PKU) are playing a part in a campaign urging the Government to make the funding available for a drug that represents their loved one's only chance at a semblance of a normal life.

Tralee woman Bernadette Gilroy addressed the Dáil on the issue a fortnight ago, fighting for her six-year-old daughter Maria's chances amid a condition that forces sufferers to undergo an alarming restrictive diet keeping the build-up of a common protein they can't break down. Its build-up can ultimately lead to brain damage.

"How is a patient's need for treatment whilst struggling to manage PKU any different to a patient's need for treatment for any other rare genetic disorder?" Biomarin posed in its statement, adding: "BioMarin was disappointed to receive a negative opinion from the NCPE". Kuvan is currently reimbursed in more than 20 countries in the European Union including Bulgaria, Latvia, Estonia, Lithuania, Slovakia and Slovenia, in addition to Germany, France, Spain and Italy.