Miglustat: 6 agencies, 2003–2025
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
On the same population the two systems disagree outright: SMC (Scotland) recorded recommended for “Moderate type 1 Gaucher disease”, while AWMSG (Wales) recorded not recommended for “For the oral treatment of adult patients with mild to moderate type 1 Gaucher disease.”.
The file has been open for more than a decade: 21.7 years separate the first record, 28 Oct 2003 at TLV (Sweden), from the most recent, 10 Jun 2025 at PHARMAC (New Zealand).
AWMSG (Wales) changed its position: not recommended on 27 Jul 2012, recommended by 9 Oct 2024, after a resubmission.
No assessment is on file from PBAC (Australia) and G-BA (Germany), each among the eight highest-volume agencies here.
Not every gap is a refusal: for miglustat, 1 submission was never made — an outcome set by the company rather than the assessor.
The file is active: 5 of 21 records date from 2023 or later.
4 distinct indication wordings are recorded for miglustat by the agencies that label one in English (AWMSG and SMC).
TLV (Sweden) was first to assess miglustat, on 28 Oct 2003; CIPM (Spain) did not open a file until 23 Oct 2023 — an unusually long tail of 20 years between the earliest and latest system to look at it.
The record arrives in waves: 5 separate years each carry two or more decisions, spread across 13 years in total.
6 of the 6 agencies here assessed miglustat more than once, HAS (France) most often at 9 entries — repeat review is the norm for this molecule rather than the exception.
6 of the 18 agencies in this archive hold a record for miglustat.
The full ledger for miglustat reads 14 recommended, 2 other, 2 not recommended and 2 restricted, with 1 in other categories.
Decisions on file
| Agency | Date | Indication as recorded | Outcome | Source |
|---|---|---|---|---|
| PHARMAC (New Zealand) | 10 Jun 2025 | Niemann Pick Type C | PTAC advice: No Formal Recommendation | source |
| AWMSG (Wales) | 9 Oct 2024 | For the treatment of progressive neurological manifestations in adult patients and paediatric patients with Niemann-Pick… | recommended (directed) | source |
| CIPM (Spain) | 23 Oct 2023 | Indicación objeto de este expediente : Opfolda (miglustat) es un estabilizador enzimático de la cipaglucosidasa alfa par… | not financed by the SNS (financed 2023-12) | source |
| HAS (France) | 18 Oct 2023 | Listing (Transparency Committee) | SMR low · ASMR V | source |
| TLV (Sweden) | 9 Mar 2012 | Zavesca kvarstår i högkostnadsskyddet med begränsning | restricted subsidy | source |
16 further decisions for miglustat, across 6 agencies, are not shown here. They link to 14 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
- 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: 21 decisions for miglustat from 6 agencies, 2003 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.