New recommendation for PHARMAC to fund sapropterin for PKU patients
The Medicine sapropterin has been recommended to be funded by PHARMACs Pharmacology and Therapeutics Advisory Committee (PTAC).
Sapropterin is a treatment for a rare metabolic condition phenylketonuria (PKU), which is one of the disorders screened for at birth. PKU results in an inability for the body to process the amino acid phenylalanine causing accumulation. Phenylalanine is difficult to avoid as it is in the majority of foods that people regularly eat including milk, dairy products, meat, fish, chicken, eggs, beans, and nuts. PKU that is not well managed can lead to intellectual disability, seizures, behavioural problems and mental disorders.
Sapropterin works to reduce the levels of phenylalanine in the body, sapropterin has been recommended for funding for those people with PKU that demonstrate a positive response to treatment and are at risk of cognitive impairment.
Te Arai BioFarma has a Medsafe registered sapropterin (Product Information available here)
The complete PTAC minutes are available here : 9 and 10 August 2018
Further information on PKU is available on the following links:
PKU New Zealand Charitable Trust firstname.lastname@example.org
For any queries regarding rare diseases please visit Rare Disorders NZ