The Te Arai BioFarma pharmaceutical product sapropterin has been registered by Medsafe for supply to New Zealanders. Registration occurred during international PKU awareness month.
Sapropterin has been designated as an orphan drug for treating the rare metabolic condition phenylketonuria (PKU). PKU leads to an abnormally high concentration of the amino acid phenylalanine (Phe) in the blood, which can cause brain damage.
Currently PKU management includes the use of special food products at a PHARMAC funded cost of $40,000-$60,000 per person, per year.
Despite a high profile sapropterin has never been PHARMAC funded for New Zealanders. In Australia sapropterin (Biomarin Pharmaceutical Australia Pty Ltd) is funded by the PBS at a cost of approximately $1,000 NZD per 30 tablets.
Te Arai BioFarma COO Scott Lissington was excited that the first product requirement for patient access is complete. “There are approximately 120 New Zealanders that experience PKU, with most of them being children and adolescents. The regulatory approval of our sapropterin is an important step to funded availability of sapropterin to improve the lives of these young people, as well as the lives of their families.”
The 2017 Guidelines on the management of PKU recommend that all people with PKU are tested for sapropterin response. This can be achieved either by a 48 hour loading dose test or by genotyping. People treated to reduce Phe levels are recommended to maintain certain levels to avoid negative health outcomes:
Children to the age of 12 years 120-360 umol/L
During pregnancy 120-260 umol/L
Children over 12 years and adults 120-600 umol/L
People with PKU and their families are highly motivated to improve their health and access to new medicines. Recently the Australian drug funding agency (PBAC) received a record number of public submissions in relation to the funding of sapropterin.
Sapropterin is one of a number of products to treat rare conditions that Te Arai BioFarma is currently working to make available.
For more information on PKU you can visit here
Or contact the National Metabolic Service www.starship.org.nz
For any queries including supply of sapropterin please contact firstname.lastname@example.org