Irish children risk losing life-changing drug as HSE won't reimburse it
The company that produces the drug Vimizim, which costs €400,000 a year for an adult, has said it will appeal the HSE's decision not to reimburse it.
In a statement on the refusal, which has left at least two Irish children at risk of losing access to the life-changing drug, the company also confirmed it has been in talks with the HSE about the drug since 2015.
Vimizim treats Morquio, an ultra-rare genetic condition caused by a failure to produce an enzyme. It results in skeletons not growing and causes associated severe joint pain and fatigue. The enzyme replacement therapy, Vimizim, is the only drug known to treat the condition.
Cezy Focsa (11) is one of the two girls affected. Her mother Elena said: "This is a progressive and damaging condition. I do not know how she will be without the medicine. It gives her stamina and energy and it is our only chance for her. We do not have another chance."
Elena found out about the decision from her daughter's consultant and said: "I was angry and frustrated and disappointed."
The family, who live in Kilkenny, were delighted that Cezy was one of two Irish children included in the trials for Vimizim which began in September 2011.
It was approved by the European Medicines Agency in 2014 and the manufacturer BioMarin continued to provide it to the girls free of charge on compassionate grounds.
In the meantime, they were waiting for Vimizim to be assessed by the National Centre for Pharmacoeconomics (NCPE), which decided in December not to recommend it for reimbursement.
The HSE said it had informed BioMarin it would not be reimbursing the company for the drug.