paroxysmal nocturnal haemoglobinuria
Haematology · 2008–2025
Every decision
| Medicine | Meeting | Outcome | Consumer input |
|---|---|---|---|
| pegcetacoplan Treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who are either treatment-naïve to complement 5 inhibitors (C5is) or currently… | May 2025 | Recommended with restriction | none received |
| ravulizumab Paediatric patients with paroxysmal nocturnal haemoglobinuria (PNH). | Jul 2023 | Recommended with restriction | none received |
| pegcetacoplan Treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH) who have inadequate clinical response or are intolerant to C5 inhibitor treatment. | Jul 2022 | Recommended with restriction | none received |
| pegcetacoplan Treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH) who have inadequate clinical response to C5 inhibitor treatment. | Mar 2022 | Not recommended | 1 individuals PNH Support Association of Australia |
| ravulizumab Treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH). | Jul 2021 | Recommended with restriction | 52 individuals PNH Support Association of Australia |
| ravulizumab Treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH). | Jul 2020 | Not recommended | 36 individuals PNH Support Association of Australia |
| eculizumab Treatment of paroxysmal nocturnal haemoglobinuria (PNH) to reduce haemolysis in patients with classic PNH who meet specified eligibility criteria. | Jul 2010 | Recommended | no section in document |
| eculizumab Treatment of paroxysmal nocturnal haemoglobinuria (PNH) to reduce haemolysis. | Mar 2009 | Recommended | no section in document |
| eculizumab Treatment of patients with paroxysmal nocturnal haemoglobinuria (PNH) to reduce haemolysis. | Jul 2008 | Not recommended | no section in document |
Who spoke
PNH Support Association of Australia
What the PBS pays
$188M in government benefit over 2024–25, across 3,741 services.
This is spend on the medicines considered for this condition, not spend on the disease. A medicine used for several conditions is counted in full under each, so figures across conditions add to more than national PBS expenditure.
| Medicine | Government benefit |
|---|---|
| ravulizumab | $105.7M |
| Eculizumab | $37.1M |
| eculizumab | $37.1M |
| pegcetacoplan | $8.4M |
Appraised elsewhere
NICE, in England and Wales, has appraised 5 technologies for this condition, recommending 5 . 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 |
|---|---|---|---|
| crovalimabCrovalimab for treating paroxysmal nocturnal haemoglobinuria in people 12 years and over | TA1019 | recommended | 2024-11 |
| danicopanDanicopan with ravulizumab or eculizumab for treating paroxysmal nocturnal haemoglobinuria | TA1010 | recommended restricted | 2024-10 |
| iptacopanIptacopan for treating paroxysmal nocturnal haemoglobinuria | TA1000 | recommended restricted | 2024-09 |
| pegcetacoplanPegcetacoplan for treating paroxysmal nocturnal haemoglobinuria | TA778 | recommended restricted | 2022-03 |
| ravulizumabRavulizumab for treating paroxysmal nocturnal haemoglobinuria | TA698 | recommended | 2021-05 |
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 |
|---|---|---|
| danicopanVOYDEYA 50 + 100 mg film-coated tablets, composite bottle pack | 2024 | 3 |
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: 3 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.