Danicopan: clinical use

Prescribing considerations

• Initiation should be by a healthcare professional experienced in haematological disorders.
• Use only as an add-on to ravulizumab or eculizumab; efficacy as monotherapy is not established.
• Confirm meningococcal vaccination status and arrange temporary antibiotic prophylaxis when vaccination has not been completed sufficiently in advance.
• Use caution with active systemic infection, severe renal impairment or body weight below 60 kg.
• Severe hepatic impairment has not been studied and use is not recommended.
• Plan gradual withdrawal rather than abrupt discontinuation.

Contraindications and cautions

• Hypersensitivity to danicopan or any excipient.
• Unresolved Neisseria meningitidis infection at treatment initiation.
• Absence of current meningococcal vaccination unless appropriate antibiotic prophylaxis is provided.

Monitoring

• Check liver enzymes before treatment and continue routine chemistry monitoring during PNH care.
• Monitor for early meningococcal infection, sepsis and other serious infections.
• Monitor adverse effects more closely with severe renal impairment or body weight below 60 kg.
• Assess haemolysis, haemoglobin response and ongoing need for add-on treatment.

Clinical pharmacology

Danicopan is a reversible, selective factor D inhibitor that suppresses the alternative complement pathway while leaving classical and lectin pathway activity intact. It is highly protein-bound, extensively metabolised mainly through non-CYP pathways and eliminated predominantly in faeces.

Tablet

Route: Oral

Strengths: 50 mg, 100 mg