EliglustatCerdelga
Treatment of Gaucher disease type 1 (GD1) in adult patients aged ≥18 years with at least one GD1-related disease manifestation (skeletal disease, haematological complications, or gastrointestinal complications due to enlarged liver or spleen).
Decision on record
- Meeting Jul 2015 Not recommended Gaucher Disease
Access path
- TGA registered · Cerdelga
TGA label narrower than the PBS population
- Jul 2015Not recommended
vs enzyme replacement therapy (imiglucerase or…
From the public summary
7.1 The PBAC rejected the request to list eliglustat on the PBS for the treatment of Gaucher Disease type 1 on the basis that the results of the direct randomised trial (ENCORE) suggested inferiority, and that clinically important inferiority could not be excluded with confidence. 13PSD · Jul 2015
7.2 The PBAC accepted that the nominated comparators, imiglucerase and velaglucerase-alfa, were appropriate.PSD · Jul 2015
6.27 A cost-minimisation analysis against imiglucerase (as a representative for ERT) was presented by the submission.PSD · Jul 2015
6.28 The equi-effective doses were estimated as ERT (imiglucerase or velaglucerase-alfa)PSD · Jul 2015
6.24 The submission described eliglustat as non-inferior in terms of comparative effectiveness and non-inferior in terms of comparative safety over imiglucerase.PSD · Jul 2015
These claims were not adequately supported as: The definition of a “stable” response in ENCORE was broader and more relaxed compared to Kishnani et al 2009 and the non-inferiority margin of 25% assumed that eliglustat is 10% worse than imiglucerase, which may not be reasonable for a non-inferiority claim as no reasonable justification for a MCID was provided in either the submission or the PSCR.PSD · Jul 2015
Cost-effectiveness
Cost-minimisation analysis; no ICER calculated as this was a cost-minimisation comparison against ERT.
Decision context
PopulationAdults aged ≥18 years with confirmed diagnosis of Gaucher disease type 1 and at least one GD1-related disease manifestation (splenomegaly, hepatomegaly, anaemia, thrombocytopenia, or skeletal disease) who are CYP2D6 poor, intermediate or extensive metabolisers.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Jul 2015 | Not recommended | enzyme replacement therapy (imiglucerase or velaglucerase-alfa) | — | RCT · Maintenance of response in composite outcome (haemoglobin level, platelet count, spleen volume and liver volume) |
Clinical evidence
| Trial | Phase | N | Primary outcome | Status |
|---|---|---|---|---|
| ENCORE | Ph 3 | 160 | Percentage of Participants Who Remained Stable for 52 Weeks During the Primary Analysis Pe… | completed |
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to patients with documented GD1-related manifestations and specific CYP2D6 metaboliser status; TGA label applies to all adult GD1 patients.