Sapropterin: where the agencies disagree
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
AOTMIT (Poland), HAS (France) and NICE (England), among others, recommended or restricted sapropterin, while CIPM (Spain) and SMC (Scotland) did not recommend it.
The file has been open for more than a decade: 15.8 years separate the first record, 4 Feb 2009 at HAS (France), from the most recent, 21 Nov 2024 at TLV (Sweden).
TLV (Sweden) changed its position: not recommended on 23 Dec 2009, restricted by 21 Nov 2024, after a resubmission.
No assessment is on file from G-BA (Germany), which is among the eight highest-volume agencies here.
The file is active: 5 of 23 records date from 2023 or later.
6 distinct indication wordings are recorded for sapropterin by the agencies that label one in English (AWMSG, NICE, PBAC and SMC).
5 of these 23 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 sapropterin, on 4 Feb 2009; CIPM (Spain) did not open a file until 15 Jun 2023 — an unusually long tail of 14.3 years between the earliest and latest system to look at it.
The record arrives in waves: 7 separate years each carry two or more decisions, spread across 12 years in total.
Restriction is doing real work here: 5 of 20 decisive outcomes narrowed sapropterin to a subgroup rather than accepting or refusing it outright, at NICE (England), PHARMAC (New Zealand) and TLV (Sweden).
6 of the 11 agencies here assessed sapropterin more than once, PBAC (Australia) most often at 5 entries — repeat review is the norm for this molecule rather than the exception.
11 of the 18 agencies in this archive hold a record for sapropterin.
The full ledger for sapropterin reads 10 recommended, 5 restricted, 5 not recommended and 2 deferred, with 1 in other categories.
Decisions on file
| Agency | Date | Indication as recorded | Outcome | Source |
|---|---|---|---|---|
| TLV (Sweden) | 21 Nov 2024 | Ny beredningsform av Sapropterin Dipharma ingår i högkostnadsskyddet | restricted subsidy | source |
| AOTMIT (Poland) | 9 Aug 2024 | Kuvan reimbursement recommendation | recommended | source |
| CIPM (Spain) | 15 Jun 2023 | Indicacio nes terapéutica s autorizada s: Sapropterina Dipharma está indicado para el tratamiento de la hiperfenilalanin… | not financed by the SNS · art. 92 d | source |
| NYE METODER (Norway) | 21 May 2023 | Behandling av hyperfenylalaninemi (HPA) hos voksne og barn i alle aldre med fenylketonuri (PKU) (se full indikasjonsteks… | introduced | source |
| HAS (France) | 22 Feb 2023 | Listing (Transparency Committee) | SMR substantial · ASMR V | source |
18 further decisions for sapropterin, across 9 agencies, are not shown here. They link to 12 further source documents.
Related molecules
- teduglutide — 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
- velmanase alfa — same ATC class A16A — Other alimentary tract and metabolism
Coverage: 23 decisions for sapropterin from 11 agencies, 2009 to 2024. 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.