NitisinoneNITYR
Treatment of patients with hereditary tyrosinaemia type 1 (HT-1).
Decisions on record
- Meeting Nov 2025 Recommended Hereditary tyrosinaemia type 1 no PSD
- Meeting Jul 2025 Deferred Hereditary tyrosinaemia type 1 no PSD
- Meeting Jul 2015 Not recommended Tyrosinaemia
- Meeting Nov 2014 Deferred 2 mg capsule, 60 5 mg capsule, 60 10 mg capsule, 60 Orfadin® A.Menarini Australia Pty Ltd New Listing (Major submission) Hereditary tyrosinaemia type 1 Section 100 (Highly Specialised Drugs) listing for the treatment of hereditary tyrosinaemia type 1 (HT-1). On the basis of one single-arm study pres
Access path
- TGA registered · NITYR
- Nov 2014Deferred
vs standard medical management without nitisinone (diet alone)
- Jul 2015Not recommended
uncertain and unacceptably high estimate of cost effectiveness, high cost of nitisinone, unclear safety profile…
From the public summary
6.1 The PBAC rejected the request to list nitisinone on the PBS for the treatment of HT-1 on the basis of an uncertain and unacceptably high estimate of cost effectiveness.PSD · Jul 2015
6.2 The PBAC recalled that in November 2014, it noted the clinical need for nitisinone for the treatment of HT-1 and that patients are currently being treated with the drug through hospitals.PSD · Jul 2015
5.11 In November 2014 the PBAC recommended that if treatment with nitisinone is commenced prior to the development of clinical symptoms (i.e. prior to 1 month of age), the results of the Larochelle 2012 study would be considered relevant. It was noted that such a scenario would occur if succinylacetone (SA) screening (a highly sensitive and specific test for HT-1) for all newborns were to be implemented.PSD · Jul 2015
According to the Larochelle study, the introduction of SA testing would result in an incremental cost per life year-gained for early nitisinone treatment of $105,000 – $200,000 and $105,000 – $200,000 per quality adjusted life year (QALY) gained.PSD · Jul 2015
5.10 The PBAC recalled that the November 2014 submission described nitisinone plus diet therapy as superior in terms of comparative effectiveness and superior in terms of comparative safety over diet alone. At the July meeting the PBAC reiterated its consideration from November 2014 that this claim was reasonable in terms of effectiveness but not reasonable in terms of safety.PSD · Jul 2015
5.2 The PBAC noted and welcomed the input from individuals (18) and an organisation (1) via the Consumer Comments facility on the PBS website. The comments described a range of benefits of treatment with nitisinone including the fact that it is a reliable and effective treatment which enables patients to live a longer life than would otherwise be possible.PSD · Jul 2015
Cost-effectiveness
ICER stated for early nitisinone treatment based on Larochelle 2012 trial ($105,000–$200,000 per QALY). PBAC also noted that the ICER for continuation of current screening practices exceeded $200,000 per QALY. PBAC considered both ICERs unacceptably high.
The PBAC considered that the ICERs presented were unacceptably high, regardless of SA screening status. PBAC · 2015
Decision context
PopulationPatients with a confirmed clinical diagnosis of hereditary tyrosinaemia type 1 based on detection of succinylacetone in urine and/or blood, to be treated in combination with dietary restriction of tyrosine and phenylalanine at a centre with experience in metabolic disorders.
Risk sharingSponsor indicated willingness to enter into discussion with PBAC on a Risk Sharing Agreement and potential mechanisms to contain the risk associated with the cost of the drug to the PBS.
Why it was knocked back
- uncertain and unacceptably high estimate of cost effectiveness, high cost of nitisinone, unclear safety profile particularly regarding neurocognitive disorders, inadequately addressed prior PBAC concerns regarding cost per patient per year
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Jul 2015 | Not recommended | standard medical management without nitisinone (diet alone) | $105k–200k | Single-arm · LY |
| Nov 2014 | Deferred | standard medical management without nitisinone (diet alone) | — | Single-arm · OS |
Consumer voice
Eighteen individuals and one organisation (Metabolic Dietary Disorders Association) provided consumer input describing nitisinone as a reliable and effective treatment that extends life and improves access security. The organisation highlighted benefits including life extension, condition management, and avoiding costly liver transplants, while noting current equity and access issues.
it is a reliable and effective treatment which enables patients to live a longer life than would otherwise be possible Consumer comments · PSD