Agalsidase AlfaReplagal
Recommended Rare disease
treatment of Fabry disease, a rare X-linked genetic disorder caused by a deficiency of the enzyme alpha-galactosidase A.
1
Submissions
2009–09
On the record
—
ICER range
Not modelled
Cost basis
Access path
- TGA registered · Replagal
TGA label equal than the PBS population
- Nov 2009Recommended
vs agalsidase beta
- PBS listing
RecommendedDeferredNot recommended
Cost-effectiveness
This is a literature review document for regulatory/policy comparison of two already-listed drugs under the Life Saving Drugs Program. No economic evaluation or ICER was conducted.
Decision context
Populationpatients with Fabry disease
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Nov 2009 | Recommended | agalsidase beta | — | RCT, Registry · OS, PFS, DFS, ORR, QoL, Surrogate |
Similar precedents
Regulatory · TGA
Label equal than PBS population — Both texts describe the same indication: enzyme replacement therapy for patients with Fabry disease due to alpha-galactosidase A deficiency.