Pharmac will begin funding the drug nitisinone for New Zealanders living with the rare metabolic disorders tyrosinemia type 1 and alkaptonuria from 1 February 2026, shifting the treatment from its current case‑by‑case approval pathway to full listing on the Pharmaceutical Schedule.
The move is expected to benefit around 10 people nationwide in the first year of funding.
Pharmac’s Acting Manager of Pharmaceutical Funding, Claire Pouwels, said the decision would significantly streamline access for patients and clinicians.
“This decision will make it easier for people living with these rare conditions to access nitisinone,” Pouwels said. “It will also make it easier for clinicians to prescribe this treatment, without needing to apply through NPPA each time.”
Nitisinone is currently accessed through Pharmac’s Named Patient Pharmaceutical Assessment (NPPA) process, which requires individual clinical applications. From 2026, the medicine will be routinely available, removing administrative barriers and providing greater certainty for families and clinicians.
Clinicians have backed the decision, saying the change will simplify treatment pathways for patients with these lifelong conditions.
Rare Disorders New Zealand also previously supported the proposal, noting that a scheduled listing would reduce administrative burden and improve transparency around access criteria.
About the Conditions
Tyrosinemia type 1 and alkaptonuria are inherited metabolic disorders that affect the body’s ability to break down the amino acid tyrosine. Without treatment, toxic by‑products can accumulate, leading to serious health complications.
Nitisinone, used alongside a controlled diet, can prevent or reduce these harmful effects.
Brand Change and Support Pathways
People already receiving nitisinone through NPPA will continue treatment uninterrupted, but may need to transition to the LogixX Pharma brand from February 2026.
Pharmac says that if patients experience issues with the new brand, clinicians can apply for an alternative under the Exceptional Circumstances framework.
Government Response
Searches show no public statements from health ministers or Te Whatu Ora officials regarding this funding decision. The announcement appears to have been made solely by Pharmac, with commentary from clinicians and Rare Disorders NZ supporting the move.
