Fabry disease
Rare disease · 2009–2025
7decisions
3medicines
4carried consumer input
72individual submissions
Every decision
| Medicine | Meeting | Outcome | Consumer input |
|---|---|---|---|
| pegunigalsidase alfa Treatment of Fabry disease in adult patients with end-organ damage (renal, cardiac, cerebrovascular disease, or uncontrolled chronic pain). | Jul 2025 | Recommended with restriction | 4 individuals |
| migalastat Treatment of Fabry disease in patients aged 12 years and older with a confirmed diagnosis of Fabry disease and who have an amenable mutation. | May 2024 | Recommended | 47 individuals Fabry Australia · Genetic Alliance Australia · National Fabry Disease Foundation · Australian Pompe Association · Human Genetics Society |
| migalastat Treatment of Fabry disease in patients 16 years of age and older who have an amenable mutation. | Dec 2022 | Recommended with restriction | no section in document |
| migalastat Treatment of Fabry disease in patients aged 16 and over who have an amenable mutation. | Jul 2017 | Not recommended | 11 individuals Rare Voices Australia · Fabry Australia |
| migalastat Long-term treatment of Fabry disease in patients aged 16 years and older who have an amenable GLA mutation. | Mar 2017 | Deferred | none received |
| migalastat Long-term treatment of adult and adolescent patients 16 years and older with a confirmed diagnosis of Fabry disease (α-galactosidase A deficiency) and who have… | Nov 2017 | Not recommended | 10 individuals Fabry Australia |
| agalsidase alfa treatment of Fabry disease, a rare X-linked genetic disorder caused by a deficiency of the enzyme alpha-galactosidase A. | Nov 2009 | Recommended | no section in document |
Who spoke
Fabry Australia · Rare Voices Australia · Genetic Alliance Australia · National Fabry Disease Foundation · Australian Pompe Association · Human Genetics Society
Conditions are classified by a language model from the indication text of each submission, so an individual label here can be wrong. Consumer input is read from the document's own consumer comments section: 4 of 6 decisions since 2014 carried some. Silence means no comment reached the committee through that facility, not that nobody was affected. More on how this was built.