late-onset Pompe disease
Metabolic · 2009–2025
8decisions
2medicines
2carried consumer input
16individual submissions
Every decision
| Medicine | Meeting | Outcome | Consumer input |
|---|---|---|---|
| cipaglucosidase alfa with miglustat Treatment of adults with late-onset Pompe disease (LOPD). CIPAMIG is a combination therapy where cipaglucosidase alfa is a recombinant human GAA enzyme and… | Mar 2025 | Not recommended | 16 individuals Australian Pompe Association |
| alglucosidase alfa Treatment of patients with late-onset Pompe disease (acid alfa-glucosidase deficiency). | Mar 2013 | Recommended | unclear |
| alglucosidase alfa Long-term treatment of patients with a confirmed diagnosis of late-onset Pompe disease (acid alfa-glucosidase deficiency). | Nov 2012 | Recommended | no section in document |
| alglucosidase alfa Long-term treatment of patients with late-onset Pompe disease (acid alfa-glucosidase deficiency). | Nov 2012 | Recommended | no section in document |
| alglucosidase alfa Treatment of late-onset Pompe disease (acid alfa-glucosidase deficiency), an inherited lysosomal storage disorder characterised by progressive muscle weakness. | Jul 2011 | Deferred | unclear |
| alglucosidase alfa Late-onset Pompe disease (acid alpha-glucosidase deficiency) with symptomatic muscle weakness and/or impaired respiratory function. | Nov 2010 | Not recommended | input received |
| alglucosidase alfa Treatment of late-onset Pompe disease (acid alfa-glucosidase deficiency) in patients aged 8 years or older with confirmed diagnosis and documented enzyme… | Mar 2009 | Not recommended | no section in document |
| alglucosidase alfa Treatment of late-onset Pompe disease (acid alpha-glucosidase deficiency) in patients with significant deterioration in lung function or muscle function. | Nov 2009 | Recommended | no section in document |
Who spoke
Australian Pompe Association
Appraised elsewhere
NICE, in England and Wales, has appraised 1 technology for this condition, recommending 1 . A different committee, a different population and a different price: this is what has been looked at there, not a verdict on what happened here.
| Technology | Appraisal | Outcome | Date |
|---|---|---|---|
| cipaglucosidase alfaCipaglucosidase alfa with miglustat for treating late-onset Pompe disease | TA912 | recommended restricted | 2023-08 |
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: 1 of 1 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.