Danicopan
Hemolysis
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Drug Status
Government Approvals
US(FDA), UK(BNF)
WHO Essential Medicine
NO
Known Teratogen
No
Pharmaceutical Class
NA
Controlled Drug Substance
No
Danicopan
Summary
Danicopan is used for treating extravascular hemolysis in adults with a condition called paroxysmal nocturnal hemoglobinuria (PNH). It's typically used in combination with other medications.
Danicopan works by binding to a protein called complement Factor D in your body. This inhibits a process called the alternative complement pathway, which reduces the breakdown of red blood cells, a problem in PNH patients.
Danicopan is taken orally, typically three times a day. The usual daily dose for adults is 150 mg, which can be increased to 200 mg based on your response to the medication.
The most commonly reported side effect of Danicopan is headache, which occurs in about 11% of patients. Other side effects include vomiting and increased risk of infections.
Danicopan should not be used during breastfeeding and its use during pregnancy should be considered carefully. It's also contraindicated in patients with unresolved serious infections. It's important to get vaccinated against certain bacteria before starting treatment with Danicopan.
Indications and Purpose
How does Danicopan work?
Danicopan binds to complement Factor D and inhibits the alternative complement pathway. This action prevents the cleavage of complement Factor B, reducing the formation of the C3 convertase and subsequent hemolysis in PNH patients.
Is Danicopan effective?
Danicopan has been shown to be effective in a clinical trial for patients with paroxysmal nocturnal hemoglobinuria (PNH) who have clinically significant extravascular hemolysis. It demonstrated a significant increase in hemoglobin levels and improvement in fatigue scores compared to placebo.
What is Danicopan?
Danicopan is used as an add-on therapy for treating extravascular hemolysis in adults with paroxysmal nocturnal hemoglobinuria (PNH). It works by inhibiting complement Factor D, which reduces the activation of the alternative complement pathway, thereby decreasing hemolysis.
Directions for Use
For how long do I take Danicopan?
Danicopan is typically used as a long-term treatment for paroxysmal nocturnal hemoglobinuria (PNH) in combination with other medications. The duration of use depends on the patient's response and the treating physician's guidance.
How do I take Danicopan?
Danicopan should be taken orally three times a day, with or without food. There are no specific food restrictions mentioned, but it is important to follow your healthcare provider's instructions.
How long does it take for Danicopan to start working?
Danicopan reaches steady-state concentration in approximately 2 days, and its effects on hemoglobin levels and fatigue may be observed within a few weeks of starting treatment.
How should I store Danicopan?
Store Danicopan in the original container at room temperature, between 68°F and 77°F (20°C and 25°C). Keep it away from children and do not use it after the expiration date.
What is the usual dose of Danicopan?
The usual daily dose for adults is 150 mg taken three times a day, which can be increased to 200 mg three times a day based on clinical response. The safety and effectiveness of Danicopan in children have not been established.
Warnings and Precautions
Can Danicopan be taken safely while breastfeeding?
Danicopan is present in animal milk, and it is likely to be present in human milk. Due to the potential for serious adverse reactions in the breastfed child, breastfeeding is not recommended during treatment with Danicopan and for 3 days after the last dose.
Can Danicopan be taken safely while pregnant?
There are no available data on Danicopan use in pregnant individuals. Animal studies have shown no adverse developmental effects at high exposures. The use of Danicopan in pregnancy should be considered only after assessing the risks and benefits.
Can I take Danicopan with other prescription drugs?
Danicopan is a BCRP and P-gp inhibitor. It can increase the plasma concentrations of drugs that are substrates of these proteins, such as rosuvastatin and fexofenadine. Dose adjustments may be necessary for these drugs.
Is Danicopan safe for the elderly?
There is limited experience with Danicopan in patients aged 65 and older. No specific dose adjustment is required for elderly patients, but they should be monitored closely for any adverse effects.
Who should avoid taking Danicopan?
Danicopan increases the risk of serious infections caused by encapsulated bacteria. Patients must be vaccinated against these bacteria before starting treatment. It is contraindicated in patients with unresolved serious infections caused by encapsulated bacteria.
