Paroxysmal nocturnal haemoglobinuria (PNH) Program in Pharmaceutical Benefits Scheme (PBS) 012-22022230

PNH restriction and item codes

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$\\INTERNAL.DEPT.LOCAL\Shared\NAT\SERDELEXCEL\WORKPRODIMP\Operation Blueprint Migration\RDT Release Icons\32w\icon-hidden-attachment.png$ PBS PNH restriction and item codes

Eculizumab PNH dosing schedule for patients under 18 years

Table 1

Body weight	Induction phase	Maintenance phase
40kg and over	600mg weekly x 4 doses	900mg at week 5, then 900mg every 2 weeks
30kg to less than 40kg	600mg weekly x 2 doses	900mg at week 3, then 900mg every 2 weeks
20kg to less than 30kg	600mg weekly x 2 doses	600mg at week 3, then 600mg every 2 weeks
10kg to less than 20kg	600mg weekly x 1 dose	300mg at week 2, then 300mg every 2 weeks
5kg to less than 10kg	300mg weekly x 1 dose	300mg at week 2, then 300mg every 3 weeks

Ravulizumab PNH loading dose and vial calculator (adults only)

Table 2

Body weight	Loading dose	Number of 300mg vials	Number of 1100mg vials
40kg to less than 60kg	2400mg	8	0
60kg to less than 100kg	2700mg	9	0
100kg and over	3000mg	10	0

Ravulizumab PNH maintenance dose and vial calculator (adults only)

Table 3

Body weight	Maintenance phase	Number of 300mg vials	Number of 1100mg vials
40kg to less than 60kg	3000mg	10	0
60kg to less than 100kg	3300mg	0	3
100kg and over	3600mg	1	3

Ravulizumab PNH loading dose and vial calculator (paediatric patients who weigh <40kg)

Table 4

Body weight	Loading dose	Number of 300mg vials	Number of 1100mg vials
5kg to less than 10kg	600mg	2	0
10kg to less than 20kg	600mg	2	0
20kg to less than 30kg	900mg	3	0
30kg to less than 40kg	1200mg	4	0

Ravulizumab PNH maintenance dose and vial calculator (paediatric patients who weigh <40kg)

Table 5

Body weight	Maintenance phase	Number of 300mg vials	Number of 1100mg vials
5kg to less than 10kg	300mg	1	0
10kg to less than 20kg	600mg	2	0
20kg to less than 30kg	2100mg	7	0
30kg to less than 40kg	2700mg	9	0

Pegcetacoplan PNH dosing schedule (adults only)

Table 6: Pegcetacoplan 1080mg/20mL (54mg/mL) SC infusion

Dose	Frequency	Quantity
Recommended dose	1080mg (1 vial) twice weekly	8 or 9 vials per month
Dose adjustment (for patient LDH > 2*ULN )	1080mg (1 vial) every 3 days	10 vials per month

Contact details

PBS Complex Drugs Programs

Forms

Paroxysmal nocturnal haemoglobinuria - eculizumab or ravulizumab - initial, switching, returning or balance of supply authority application (PB327)

Paroxysmal nocturnal haemoglobinuria - eculizumab or ravulizumab - initial grandfather authority application (PB329)

Paroxysmal nocturnal haemoglobinuria - eculizumab or ravulizumab - continuing authority application (PB328)

Paroxysmal nocturnal haemoglobinuria - pegcetacoplan - initial authority application (PB343)

Paroxysmal nocturnal haemoglobinuria - pegcetacoplan - continuing or returning authority application (PB344)

Services Australia website

Paroxysmal nocturnal haemoglobinuria

External websites

PBS schedule for Paroxysmal nocturnal haemoglobinuria

FAQs for Service Officers

Table 1: see Table 2 in Processing Complex Authority Required Listings for FAQs about all Complex programs.

Item	Description
1	Can PBS-subsidised pegcetacoplan for the treatment of paroxysmal nocturnal haemoglobinuria (PNH) be prescribed for patients under 18? No. Pegcetacoplan is PBS-subsidised for adult patients (aged 18 years or older) only.
2	Is there an age restriction for PBS-subsidised eculizumab or ravulizumab to treat PNH? No. Prescribers can seek approval to treat patients of any age.
3	Which of the 3 PNH treatments (i.e. ravulizumab, eculizumab & pegcetacoplan) is suitable for patients who are pregnant or planning for pregnancy? Eculizumab.
4	Which of the 3 PNH treatments (i.e. ravulizumab, eculizumab & pegcetacoplan) is listed under the Australian Government’s Life Saving Drugs Program (LSDP)? Eculizumab.
5	When will the grandfather restriction of ravulizumab and eculizumab expire? The grandfather restriction for both drugs will stay in place for 5 years after being listed - longer than the standard 12-month timeframe. Both drugs were listed on 1/03/2022 and their grandfather restriction is expected to cease around 1/03/2027.
6	Do patients changing from ravulizumab to eculizumab for family planning purposes have to return to ravulizumab? No, they don’t have to but can choose to do so.
7	Are pathology results required for the continuing PBS-subsidised PNH treatment with ravulizumab or eculizumab? Pathology results are required for the first continuing application and must be submitted within 2 weeks of the initial application and can be the same results that were provided with the initial request. Pathology results are not required for subsequent continuing applications.
8	Can replacement or top-up prescriptions be approved using Balance of Supply (BoS) item codes? No. There are no BoS item codes. Replacement and top-up prescriptions will need to be entered under previously approved item code and restriction.
9	Where are the dosing schedules for PNH located? On the code sheet and OB. You can find dosing tables on either the Master code sheet or the PNH individual code sheet, as well as under the Resources tab on the OB page.
10	What other PBS-subsidised condition are ravulizumab and eculizumab listed to treat? Atypical haemolytic uraemic syndrome (aHUS). Refer all requests for aHUS to a pharmaceutical adviser.

Operational Information