PNH Treatment
Complement Inhibitor Treatments
Complement inhibitors target the part of the immune system that attacks PNH red blood cells. These treatments reduce haemolysis, improve anaemia, and lower the risk of complications. All complement inhibitor treatments are prescribed and monitored with input from the National PNH Service.
Complement inhibitors significantly reduce the body’s ability to fight Neisseria meningitidis (meningococcus), the bacteria that cause meningococcal meningitis and sepsis. Meningococcal infections can be dangerous as they can develop very quickly and early symptoms, such as fever, headache and feeling unwell, can be difficult to spot. Patients are usually vaccinated against meningococcus and may receive antibiotics as a precaution, but the risk is still higher than in the general population.
| Drug (Brand) | Drug Company | Administration | Controls IVH? | Controls EVH? |
|---|---|---|---|---|
| Eculizumab (Soliris) | Alexion | Intravenous (IV) infusion every 2 weeks | Yes | No |
| Ravulizumab (Ultomiris) | Alexion | IV infusion every 8 weeks | Yes | No |
| Iptacopan (Fabhalta) | Novartis | Oral capsule twice daily | Yes | Yes |
| Pegcetacoplan (Aspaveli) | Sobi | Subcutaneous injection (twice weekly) | Yes | Yes |
| Crovalimab (PiaSky) | Roche | Subcutaneous injection (monthly) | Yes | No |
| Danicopan (Voydeya) | AstraZeneca via Alexion | Oral tablets, taken twice or three times daily, in combination with an existing C5 inhibitor | Yes – through C5 inhibitor | Yes |
Available Complement Inhibitor Treatments in Scotland
Detailed administration guidelines for each of the drugs and further information on meningococcal infection can be found on the PNH National Service website
Click a drug name in the table to view more information about the drug.
Detailed administration guidelines for each of these drugs and further information on meningococcal infection can be found on the PNH National Service website
Learn MoreEculizumab (Soliris)
Eculizumab was the first complement inhibitor used in Scotland. It stops intravascular haemolysis (IVH) but not extravascular haemolysis (EVH) meaning some patients require further treatment to stop anaemia. It is given as an infusion every two weeks by a nurse either at home or in hospital. It is not recommended for PNH in Scotland meaning it is not routinely funded, or part of standard NHS Scotland PNH prescribing, but access for patients is possible through individual funding requests.
Ravulizumab (Ultomiris)
Ravulizumab is a longer-acting complement inhibitor. It has similar efficacy to Eculizumab but with less frequent dosing. It is given as an infusion every eight weeks by a nurse either at home or in hospital. It is accepted for restricted use by NHS Scotland meaning it is prescribed under the advice of the National PNH Service for adults with PNH who have clinical haemolysis.
Iptacopan (Fabhalta)
Iptacopan is an oral treatment that inhibits both intravascular haemolysis (IVH) and extravascular haemolysis (EVH). It is in tablet form which means it does not require infusions or hospital visits for administration. Iptacopan is accepted for restricted use by NHS Scotland, under the advice of the National PNH Service, for adults with PNH who have evidence of haemolytic anaemia.
Pegcetacoplan (Aspaveli)
Pegcetacoplan is a complement inhibitor that inhibits both intravascular haemolysis (IVH) and extravascular haemolysis (EVH). It is given by subcutaneous injection (under the skin) using an infusion pump. It is usually administered twice weekly, and many patients are trained to self-inject at home. Pegcetacoplan is accepted for restricted use in Scotland for adults with PNH who are anaemic after more than 3 months on non-EVH preventing inhibitors.
Crovalimab (PiaSky)
Crovalimab is a complement inhibitor which can stop intravascular haemolysis (IVH) but not extravascular haemolysis (EVH). It is given by subcutaneous injection (under the skin) which patients can be trained to administer themselves. After initial loading doses, it is usually administered once every 4 weeks. It is approved for restricted use in Scotland under the advice of the National PNH Service.
Danicopan (Voydeya)
Danicopan is an oral complement inhibitor which stops extravascular haemolysis (EVH). It is an add-on therapy taken in combination with ongoing infusions or injection therapies when anaemia persists despite good control of intravascular haemolysis. Danicopan is accepted for restricted use in Scotland as an add-on therapy for those with residual haemolytic anaemia.
Related Topics
What is PNH?
A helpful guide on Paroxysmal Nocturnal Hemoglobinuria (PNH) including common PNH terminology and symptoms.
Find Out MorePNH Care in Scotland
PNH care in Scotland is based on a shared-care model. Find out more abut PNH care in Scotland on this page.
Find Out MorePNH Treatment
There are a range of PNH treatment options available in Scotland from supportive care to advanced complement inhibitors.
Find Out More