hypophosphatasia
Metabolic · 2017–2018
Every decision
| Medicine | Meeting | Outcome | Consumer input |
|---|---|---|---|
| asfotase alfa rch Treatment of patients with juvenile-onset hypophosphatasia (HPP; onset between 6 months and 17 years). | Mar 2018 | Not recommended | 55 individuals Soft Bones Australia |
| asfotase alfa rch Treatment of patients with paediatric-onset hypophosphatasia (HPP), a rare metabolic disease presenting with defective bone mineralisation and associated… | Jul 2017 | Not recommended | 6 individuals Soft Bones Australia |
Who spoke
Soft Bones Australia
Registered, not subsidised
Medicines on the ARTG for this condition with no PBAC record at all. Registration and subsidy are separate decisions: a medicine can be legally available in Australia and never have been put to the committee.
| Medicine | On the ARTG since | Registrations |
|---|---|---|
| asfotase alfaSTRENSIQ asfotase alfa rch 12 mg/0.30 ml solution for injection vial | 2016 | 5 |
The ARTG export holds current registrations only, so the year is the earliest entry still on the register rather than a first-registration date. The condition here comes from the registered label, which is usually broader than the population a sponsor asks to have subsidised.
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: 2 of 2 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.