Danicopan at a glance
Medicine class
Complement factor D inhibitor
Licensed role
Add-on treatment for residual haemolytic anaemia in adults with PNH
How it is taken
Film-coated tablets taken by mouth with a meal or snack
Important protection
Meningococcal vaccination and infection precautions are required
What is Danicopan used for?
Danicopan has a specific licensed add-on role in PNH.
Treatment of adults with PNH who have residual haemolytic anaemia despite ravulizumab or eculizumab. It is continued alongside that C5 inhibitor, not used alone.
How does Danicopan work?
Danicopan reversibly blocks complement factor D, reducing alternative complement pathway activation and C3 fragment deposition on PNH red blood cells. This helps limit continuing red-cell destruction despite C5 inhibition; the classical and lectin complement pathways remain active.
Danicopan preparations and strengths
Tablet
Route: Oral
Strengths: 50 mg, 100 mg
How to take Danicopan
Follow the specialist’s prescription and continue ravulizumab or eculizumab as directed. Do not stop danicopan without specialist advice because withdrawal should be gradual.
Take the film-coated tablets with a meal or snack.
Swallow the prescribed tablets by mouth.
Tell the specialist if severe kidney or liver problems develop.
Danicopan side effects
Common or expected effects
- Fever or raised temperature
- Headache
- Raised liver enzymes on blood tests
- Vomiting
- High blood pressure
- Pain in the arms or legs
Get urgent medical advice
- Symptoms of meningococcal infection, including fever, rash, severe headache, stiff neck, confusion or light sensitivity, need immediate medical assessment.
Danicopan in pregnancy
There are no clinical data from pregnant women. As a precaution, danicopan should preferably be avoided during pregnancy. Anyone pregnant or planning pregnancy should promptly discuss treatment with the specialist team.
Danicopan while breastfeeding
Danicopan should not be used while breastfeeding because a risk to the infant cannot be excluded. Breastfeeding should not begin until three days after treatment has ended.
Danicopan interactions
Danicopan can increase exposure to medicines transported by P-glycoprotein or BCRP. The prescriber or pharmacist should review all medicines before and during treatment.
Dabigatran, edoxaban, digoxin, fexofenadine and tacrolimus
Danicopan mildly inhibits P-glycoprotein and may increase exposure, so additional caution or monitoring may be needed.
Rosuvastatin and sulfasalazine
Danicopan inhibits BCRP and may substantially increase exposure, requiring clinical review for adverse effects or treatment adjustment.
Common questions about Danicopan
Answers are fully visible for fast scanning and source review.
Can Danicopan be used on its own for PNH?
No. Its licensed role is as an add-on to ravulizumab or eculizumab; effectiveness as monotherapy has not been established.
Why is Danicopan added to ravulizumab or eculizumab?
It targets alternative-pathway C3 activity that can continue destroying red blood cells despite C5 inhibition.
Does Danicopan cure PNH?
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.