Paroxysmal nocturnal haemoglobinuria in patients who have residual haemolytic anaemia

Active Ingredient: Danicopan

Indication for Danicopan

Population group: only adults (18 years old or older)

Therapeutic intent: Adjunct intent

Danicopan is indicated as an add-on to ravulizumab or eculizumab for the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have residual haemolytic anaemia.

For this indication, competent medicine agencies globally authorize below treatments:

150 mg 3 times daily and after a minimum of 4 weeks 150-200 mg 3 times daily

For:

Dosage regimens

Oral, 150 milligrams danicopan, 3 times daily to meals, over the duration of 4 weeks. Afterwards, oral, between 150 milligrams danicopan and 200 milligrams danicopan, 3 times daily to meals.

Detailed description

The recommended starting dose is 150 mg three times a day administered orally, approximately 8 hours apart (± 2 hours). Dose can be increased to 200 mg three times a day after a minimum of 4 weeks of treatment depending on clinical response.

Missed doses

If a dose is missed, patients should be advised to take it as soon as it is remembered unless it is almost time for the next dose in which case patients should skip the missed dose and take the medicinal product at the next regularly scheduled time. Patients should be advised not to take 2 doses or more at the same time.

Discontinuation

Due to the possibility of alanine aminotransferase (ALT) elevations after treatment cessation, if treatment is discontinued, the dose should be tapered over a 6-day period until complete cessation, as follows:

100 mg regimen: 100 mg twice a day for 3 days, followed by 100 mg once a day for 3 days.
150 mg regimen: 100 mg three times a day for 3 days, followed by 50 mg three times a day for 3 days.
200 mg regimen: 100 mg three times a day for 3 days, followed by 100 mg twice a day for 3 days.

Elderly

No dose adjustment is required in elderly patients. However, experience with danicopan in patients ≥65 years of age is limited.

Dosage considerations

It should be taken with food (meal or snack).

Active ingredient

Danicopan
Danicopan binds reversibly to complement factor D (FD) and acts as a selective inhibitor of FD function. By inhibiting FD, danicopan selectively blocks the activation of complement alternative pathway (AP), leading to prevention of the production of multiple effectors, that include C3 fragments, after AP activation. The 2 other complement pathways (classical and lectin) remain active. Danicopan’s inhibitory effect on AP activation inhibits the deposition of C3 fragments on PNH red blood cells; such deposition is a key cause of the EVH which can become clinically significant in a small subset of patients with PNH on a C5 inhibitor. Maintenance of C5 inhibition controls the lifethreatening pathophysiological consequences of terminal complement activation underlying PNH. Read more about Danicopan

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