Nitisinone: 7 agencies, 2007–2025
This page collects every recorded health technology assessment decision for one molecule across the agencies in our archive, and compares them. The comparison is the point: each agency publishes its own verdict, none publishes how that verdict sits against the others.
What the record shows
No agency in this archive has refused nitisinone: all 11 decisive outcomes across 5 agencies were positive or positive with restrictions.
The file has been open for more than a decade: 18.1 years separate the first record, 20 Jun 2007 at HAS (France), from the most recent, 2025 at PBAC (Australia).
No assessment is on file from CIPM (Spain) and G-BA (Germany), each among the eight highest-volume agencies here.
Not every gap is a refusal: for nitisinone, 1 submission was never made — an outcome set by the company rather than the assessor.
Against its own class, nitisinone is an outlier: 100% of its decisive outcomes were positive, where molecules sharing ATC class A16A average 72%.
3 distinct indication wordings are recorded for nitisinone by the agencies that label one in English (AWMSG, PBAC and SMC).
3 of these 14 records carry a year of decision but no day, so any interval quoted here is approximate to within a year.
HAS (France) was first to assess nitisinone, on 20 Jun 2007; NYE METODER (Norway) did not open a file until 28 Aug 2022 — an unusually long tail of 15.2 years between the earliest and latest system to look at it.
The record arrives in waves: 3 separate years each carry two or more decisions, spread across 10 years in total.
2 of the 7 agencies here assessed nitisinone more than once, HAS (France) most often at 6 entries — repeat review is the norm for this molecule rather than the exception.
7 of the 18 agencies in this archive hold a record for nitisinone.
The full ledger for nitisinone reads 11 recommended, 1 non-submission, 1 other and 1 advice.
Decisions on file
| Agency | Date | Indication as recorded | Outcome | Source |
|---|---|---|---|---|
| PBAC (Australia) | 2025 | Hereditary tyrosinaemia Type 1 (HT-1) | Recommended | source |
| NYE METODER (Norway) | 28 Aug 2022 | Behandling av hereditær tyrosinemi og alkaptonuri (se fulle indikasjonstekster under) | introduced | source |
| SMC (Scotland) | 13 Dec 2021 | Treatment of adult patients with alkaptonuria (AKU). | not recommended | source |
| AWMSG (Wales) | 30 Sep 2021 | Treatment of adult patients with alkaptonuria | recommended (full) | source |
| HAS (France) | 21 Jul 2021 | Indication extension | SMR low · ASMR V | source |
9 further decisions for nitisinone, across 4 agencies, are not shown here. They link to 6 further source documents.
Related molecules
- teduglutide — same ATC class A16A — Other alimentary tract and metabolism
- sapropterin — same ATC class A16A — Other alimentary tract and metabolism
- miglustat — same ATC class A16A — Other alimentary tract and metabolism
- migalastat — same ATC class A16A — Other alimentary tract and metabolism
- cerliponase alfa — same ATC class A16A — Other alimentary tract and metabolism
- givosiran — same ATC class A16A — Other alimentary tract and metabolism
Coverage: 14 decisions for nitisinone from 7 agencies, 2007 to 2025. Data to 7 Sep 2026. ATC class A16A — Other alimentary tract and metabolism. How this page is produced. Compiled by Altima Partners; queries: sales@altimapartners.co.uk.