paroxysmal nocturnal haemoglobinuria
Haematology · 2008–2025
9decisions
3medicines
3carried consumer input
89individual submissions
Every decision
| Medicine | Meeting | Outcome | Consumer input |
|---|---|---|---|
| pegcetacoplan Treatment of paroxysmal nocturnal haemoglobinuria (PNH) in adult patients who are either treatment-naïve to complement C5 inhibitors or currently treated with… | May 2025 | Recommended with restriction | none received |
| ravulizumab Paroxysmal nocturnal haemoglobinuria (PNH) in paediatric patients. Ravulizumab is a long-acting anti-C5 monoclonal antibody administered via intravenous… | 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 | Recommended with restriction | 1 individuals PNH Support Association |
| ravulizumab Treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH) who meet specified clinical criteria including evidence of haemolysis, thrombotic events,… | Jul 2021 | Recommended with restriction | 52 individuals PNH Support Association |
| ravulizumab Treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH) meeting specific clinical criteria including PNH granulocyte clone size ≥10%, LDH ≥1.5… | Jul 2020 | Not recommended | 36 individuals PNH Support Association |
| eculizumab Treatment of paroxysmal nocturnal haemoglobinuria (PNH) to reduce haemolysis in patients with classic PNH who would benefit most from treatment. | 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 paroxysmal nocturnal haemoglobinuria (PNH) to reduce haemolysis. | Jul 2008 | Not recommended | no section in document |
Who spoke
PNH Support Association
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.