Decision to fund nitisinone for people with rare inherited metabolic disorders

What we’re doing

We are pleased to announce that nitisinone will be listed on the Pharmaceutical Schedule from 1 February 2026 for people with rare inherited metabolic disorders. This medicine will be funded subject to eligibility criteria. See the eligibility criteria below for detail.

Why we’re doing this

Currently all known patients in New Zealand with tyrosinemia type 1 and alkaptonuria receive funded nitisinone through Pharmac’s Named Patient Pharmaceutical Assessment (NPPA) policy.

Listing nitisinone on the Pharmaceutical Schedule will 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. We estimate 10 people with rare disorders will access nitisinone via the Pharmaceutical Schedule in the first year of funding.

Nitisinone was included in Pharmac’s 2024/25 Annual Invitation to Tender. Following this tender process, the decision has been made to award Principal Supply Status to the Nitisinone (LogixX Pharma) brand of nitisinone from 1 July 2026.

About nitisinone

Tyrosinemia type 1 and alkaptonuria are rare inherited metabolic disorders that affect how the body breaks down tyrosine, an amino acid. In tyrosinemia type 1, harmful substances can build up and damage the liver and kidneys. In alkaptonuria, a different substance accumulates, causing damage to joints, bones, and heart valves.

Both conditions are lifelong and require ongoing treatment with nitisinone and a special diet to prevent serious health problems.

Nitisinone is used alongside dietary measures in the treatment of tyrosinemia type 1 and alkaptonuria. Nitisinone works by blocking the breakdown of tyrosine in the body, which stops the build-up of harmful substances. This reduces the negative health outcomes associated with these health conditions.

Any changes to the original proposal?

This decision was subject to a consultation letter dated 26 November 2025. No changes have been made to the proposal following consultation.

Who we think will be most 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.

Detail about this decision

The following changes will occur in Section B and Part II of Section H of the Pharmaceutical Schedule.

From 1 February 2026, nitisinone will be listed in Section B of the Pharmaceutical Schedule under Special Authority criteria. This means that from 1 February 2026, Special Authority applications will need to be submitted to access nitisinone.

People currently receiving nitisinone via NPPA will be transferred to the nitisinone Special Authority criteria from 1 February 2026. Special Authority applications will not need to be submitted for this group.

For people who are not currently receiving funded nitisinone through NPPA, a Special Authority application will need to be submitted from 1 February 2026.

From 1 July 2026 until 30 June 2028, the Nitisinone (LogixX Pharma) brand of nitisinone will have Principal Supply Status. This means that Nitisinone (LogixX Pharma) will be the main funded brand of nitisinone until 30 June 2028.

If someone cannot use the Nitisinone LogixX Pharma brand for clinical reasons, Pharmac may consider applications for an alternative brand through the Exceptional Circumstances framework.

Special Authority for Subsidy

From 1 February 2026, nitisinone will be listed in Section B of the Pharmaceutical Schedule subject to the following Special Authority criteria.

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 will be included in Part II Section H of the Pharmaceutical Schedule to allow access in Health NZ Hospitals.

Our responses to what you told us

We are grateful for the time and care people took to respond to our consultation. A summary of the key themes raised in your feedback and our responses follows.

If you have any questions about this decision, you can email us at enquiry@pharmac.govt.nz; or call our toll-free number (9 am to 5 pm, Monday to Friday) on 0800 660 050.