Due to agreed clinical need, sapropterin (Kuvan®) has joined ten other drugs on the Life Saving Drugs Programme enabling its 1st May 2014 addition to the Pharmaceutical Benefits Scheme (PBS), despite costing up to $75,000 every year for each patient.
Authority restrictions offer PBS treatment for the 1-2% of hyperphenylalaninemia patients with tetrahydrobiopterin (BH4) deficiency, but not for those with the alternate indication of phenylketonuria (PKU). There is no alternative treatment for the life threatening BH4 genetic deficiency which occurs in around 1 per million newborns, or one Australian child born every three years.
Prompt replacement of the deficit in BH4 levels with this synthetic cofactor (combined with neurotransmitter precursors) can result in normal physical, intellectual and psychomotor development for young patients. Patients should continue a restricted phenylalanine diet and regular monitoring to optimise the development of neurological function and to prevent damage.
Please review full product information before prescribing.