Proposal to fund nitisinone for people with rare inherited metabolic disorders
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What we’re proposing
We are proposing to fund nitisinone in Section B and Part II Section H of the Pharmaceutical Schedule from 1 February 2026 for people with rare inherited metabolic disorders.
Nitisinone is funded for people with tyrosinemia type 1 or alkaptonuria, two inherited metabolic disorders (IMD) through Pharmac’s exceptional circumstances framework (NPPA). This proposal would mean that from 1 February 2026 nitisinone would be listed on the Pharmaceutical Schedule and funded for people in New Zealand who need it.
Consultation closes at 5:00 pm 12 December 2025 and feedback can be emailed to Consult@Pharmac.govt.nz
What would the effect be?
From 1 February 2026, the Nitisinone (LogixX Pharma) brand of nitisinone supplied by Te Arai BioFarma Limited would be listed on the Pharmaceutical Schedule for use in the community and Health New Zealand Hospitals.
People with inherited metabolic disorders would have access to nitisinone. People with tyrosinemia type 1 or alkaptonuria who are currently receiving nitisinone through Pharmac’s exceptional circumstances framework could continue to access treatment from their pharmacy or hospital in the same way they do now. People who are receiving funded nitisinone for tyrosinemia type 1 or alkaptonuria and are using brands other than the Nitisinone (LogixX Pharma) brand would change to the Nitisinone (LogixX Pharma) brand between 1 February 2026 and 30 June 2026.
We encourage people who are receiving nitsinone and their family, whānau and caregivers to discuss this proposed change with their healthcare professional.
From 1 February 2026 healthcare professionals treating people with tyrosinemia type 1 or alkaptonuria who require nitisinone would be able to apply for funded access to the Nitisinone (LogixX Pharma) brand via Special Authority or Hospital Restriction criteria rather than needing to submit applications via Pharmac’s exceptional circumstances framework (NPPA).
From 1 July 2026 until 30 June 2028, the Nitisinone (LogixX Pharma) brand of nitisinone supplied by Te Arai BioFarma Limited would have Principal Supply Status. This means that Nitisinone (LogixX Pharma) would be the main funded brand of nitisinone until 30 June 2028.
An Alternative Brand Allowance would be available for people who are unable to change to the Nitisinone (LogixX Pharma) brand of nitsinone capsules for clinical reasons,
Who we think will be interested
- People with tyrosinemia type 1 or alkaptonuria and their whānau, family and caregivers
- Metabolic specialists and clinicians
- Dietitians, general practitioners and other healthcare professionals involved in the care of people with tyrosinemia type 1 or alkaptonuria.
- Suppliers of supplements for inherited metabolic disease.
About inherited metabolic disorders
Inherited metabolic disorders (IMDs) are genetic conditions meaning they are often inherited, present at birth, and lifelong. These disorders are often caused by differences in body proteins. These proteins can do a range of things, including:
- breaking down nutrients from food and stopping compounds building up in the body at toxic levels or
- helping produce energy for the cells of the body to work properly.
Tyrosinemia type 1 and alkaptonuria are two rare inherited metabolic disorders.
Tyrosinemia type 1 and alkaptonuria
Hereditary tyrosinemia type 1 and Alkapotonuria are both disorders of tyrosine metabolism.
People with HT1type are unable to fully breakdown fumarylacetoacetate, leading to the accumulation of a toxic metabolite called succinylacetone, that damages the liver, kidneys and causes a secondary disorder called porphyria. At the time of diagnosis, people can be extremely unwell, develop severe liver failure and liver tumours. The best outcome is early diagnosis, which is now accurately achieved through newborns screening in New Zealand.
The management of HT1 requires lifelong adherence to nitisinone, which block the pathway above where the toxic metabolites are made. As a result, patients also require a strict, low tyrosine or protein diet and special formula. More detail on the formulas or supplements available in New Zealand for the management of HT1 are available on Pharmac’s website: Supplements for Tyrosinaemia(external link). This management is life saving and often prevents the need for liver transplant.
Alkaptonuria is a rare inherited metabolic disorder of tyrosine metabolism that is cause by a block in the pathway leading to homogentisic acid accumulation. This toxic metabolite, damages connective tissue of the joints, bones, heart valves and leads to progressive joint destruction and pain, lung and heart complications.
The use of nitisinone in combination with dietary management can provide better control of the levels of tyrosine byproducts in a person’s body and improve the long-term health outcomes for people with alkaptonuria.
About nitisinone
Nitisinone is used in combination with dietary measures in the treatment of tyrosinemia type 1 and alkaptonuria. Nitisinone works by blocking thetyrosine metabolism pathway, above where the toxic metabolites of these diseases (succinylacetone and homogentisic acid respectively) accumulate. This reduces the negative health outcomes associated with these health conditions.
In HT1, dosing for children and adults is weight based. For AKU, it is a standard dose for all adults. Nitisinone is available in a capsule with three different strengths. The Medsafe datasheet and New Zealand Formulary for Children provide guidance about how to use the capsules for young children.
Why we’re proposing this
Currently all known patients in NZ with with HT1 and AKU receive funded nitisinone through Pharmac’s Exceptional Circumstances Framework (NPPA).
Nitisinone was included in Pharmac’s 2024/25 Annual Invitation to Tender as we were aware of multiple suppliers interested in supplying New Zealand. We considered that including nitisinone in this process could allow ongoing supply to be secured at competitive pricing.
As a result of this tender process, we are proposing to award Principal Supply Status to the Nitisinone (LogixX Pharma) brand of nitisinone from 1 July 2026.
The proposed listing of nitisinone on the Pharmaceutical Schedule would reduce the administrative burden for health care professionals, provide transparency around criteria for access and provide certainty of ongoing supply for the people who need it.
Details about our proposal
From 1 February 2026 the Nitisinone (LogixX Pharma) brand of nitisinone would be listed in Section B and Part II Section H of the Pharmaceutical Schedule as follows.
Pricing for the Nitisinone (LogixX Pharma) brand of nitisinone capsules would be shared following any decision on this proposal
|
Chemical |
Formulation |
Brand |
Pack size |
|---|---|---|---|
|
Nitisinone |
Cap 2 mg |
Nitisinone (LogixX Pharma) |
60 |
|
Nitisinone |
Cap 5 mg |
Nitisinone (LogixX Pharma) |
60 |
|
Nitisinone |
Cap 10 mg |
Nitisinone (LogixX Pharma) |
60 |
From 1 July 2026 until 30 June 2028, the Nitisinone (LogixX Pharma) brand of nitisinone would have Principal Supply Status. This means that Nitisinone (LogixX Pharma) would be the main funded brand of nitisinone until 30 June 2028.
Nitisinone would be listed in Section B of the Pharmaceutical Schedule under Special Authority criteria. This means that from 1 February 2026, Special Authority applications would need to be submitted to access nitisinone.
Special Authority for Subsidy
Initial application from any relevant practitioner. Approvals valid without further renewal unless notified where patient requires nitisinone for the management of inherited metabolic disorders.
Equivalent criteria would be included in Part II Section H of the Pharmaceutical Schedule to allow access in Health NZ Hospitals.
An Alternative Brand Allowance would be available for people who are unable to change to the Nitisinone (LogixX Pharma) brand of nitsinone capsules for clinical reasons.
To provide feedback
Send us an email: Consult@Pharmac.govt.nz by 5:00pm, Friday, 12 December 2025.
All feedback received before the closing date will be considered by Pharmac’s Board (or its delegate) prior to making a decision on this proposal.
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