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Metabolic disorder drug listed on the PBS


Morgan Liotta


9/05/2019 11:51:23 AM

The listing will save patients with hyperphenylalaninemia more than $90,000 a year.

Newborn baby
Newborn babies are routinely screened to detect genetic disorders.

Sapropterin dihydrochloride (sold as Kuvan) is now listed on the Pharmaceutical Benefits Scheme (PBS) for the treatment of hyperphenylalaninemia – a condition triggered by phenylketonuria (PKU), which prevents normal breakdown of protein found in some foods.
 
PKU is an inherited condition that requires lifelong management. Taking sapropterin dihydrochloride works in combination with dietary restrictions to help lower the amount of amino acid phenylalanine in the blood.
 
The Federal Government is investing $19 million to subsidise availability of the medication on the PBS, enabling patients with the condition to access it for $40.30 per script, or $6.50 with a concession card. The Government said the listing will save patients up to $90,200 a year in treatment.
 
This medicine is of particular benefit for young children with PKU, who need to start treatment early after diagnosis. Newborn screening tests are routinely performed during the first few days of life to detect whether they have inherited the disorder, which affects approximately one in 10,000 newborn babies.
 
Without treatment, children with PKU can develop progressive intellectual disability and other complications, such as hyperphenylalaninemia, from a young age. Children with PKU can grow and develop normally with treatment.



metabolic PBS Pharmaceutical Benefits Scheme


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