Listing of Pharmaceutical Benefits (NHL) - Schedule 4 part 1

Paroxysmal nocturnal haemoglobinuria (PNH)

Initial treatment (new patient)

Patient must not have received prior treatment with this drug for this condition; AND

Patient must have PNH granulocyte clone size equal to or greater than 10% within the last 3 months; AND

Patient must have experienced an inadequate response/intolerance to either: (i) Complement 5 (C5) inhibitor, (ii) pegcetacoplan; AND

Patient must have received treatment with at least one complement inhibitor for at least 3 months before initiating treatment with this drug unless intolerance of severity necessitating permanent treatment withdrawal had occurred; AND

The treatment must be the sole PBS-subsidised therapy for this condition; AND

Must be treated by a haematologist; or

Must be treated by a non-specialist medical physician who has consulted a haematologist on the patient's drug treatment details; AND

Patient must be at least 18 years of age.

The authority application must be made via the Online PBS Authorities System, or in writing via HPOS form upload or mail and must include:

(1) details of the proposed prescription(s); and

(2) a completed authority application form relevant to the indication and treatment phase (the latest version is located on the website specified in the Administrative Advice).

At the time of the authority application, details (result and date of result) of the following monitoring requirements must be provided:

(i) Haemoglobin (g/L)

(ii) Platelets (x10⁹/L)

(iii) White Cell Count (x10⁹/L)

(iv) Reticulocytes (x10⁹/L)

(v) Neutrophils (x10⁹/L)

(vi) Granulocyte clone size (%)

(vii) Lactate Dehydrogenase (LDH)

(viii) the upper limit of normal (ULN) for LDH as quoted by the reporting laboratory

(ix) the LDH:ULN ratio (in figures, rounded to one decimal place)

Listing of Pharmaceutical Benefits (NHL) - Schedule 4 part 1

Paroxysmal nocturnal haemoglobinuria (PNH)

Return from PBS-subsidised eculizumab post pregnancy or from PBS-subsidised Complement 5 (C5) inhibitor for reasons other than post pregnancy

Patient must have received prior PBS-subsidised treatment with this drug for this condition; AND

Patient must have received prior PBS-subsidised treatment with eculizumab through the 'Initial treatment - (initial 3) switching from PBS-subsidised pegcetacoplan or iptacopan for pregnancy (induction doses)' criteria; or

Patient must have received prior PBS-subsidised treatment with at least one C5 inhibitor and must be returning to iptacopan treatment for reasons other than post pregnancy; AND

Patient must have experienced clinical improvement as a result of treatment with this drug; or

Patient must have experienced a stabilisation of the condition as a result of treatment with this drug; AND

The treatment must be the sole PBS-subsidised therapy for this condition; AND

Must be treated by a haematologist; or

Must be treated by a non-specialist medical physician who has consulted a haematologist on the patient's drug treatment details; AND

Patient must be at least 18 years of age.

The authority application must be made via the Online PBS Authorities System, or in writing via HPOS form upload or mail and must include:

(1) details of the proposed prescription(s); and

(2) a completed authority application form relevant to the indication and treatment phase (the latest version is located on the website specified in the Administrative Advice).

At the time of the authority application, details (result and date of result) of the following monitoring requirements must be provided:

(i) Haemoglobin (g/L)

(ii) Platelets (x10⁹/L)

(iii) White Cell Count (x10⁹/L)

(iv) Reticulocytes (x10⁹/L)

(v) Neutrophils (x10⁹/L)

(vi) Granulocyte clone size (%)

(vii) Lactate Dehydrogenase (LDH)

(viii) the upper limit of normal (ULN) for LDH as quoted by the reporting laboratory

(ix) the LDH:ULN ratio (in figures, rounded to one decimal place)

Patients may qualify under this treatment phase more than once.

Listing of Pharmaceutical Benefits (NHL) - Schedule 4 part 1

Paroxysmal nocturnal haemoglobinuria (PNH)

Continuing treatment

Patient must have received PBS-subsidised treatment with this drug for this condition; AND

Patient must have experienced clinical improvement as a result of treatment with this drug; or

Patient must have experienced a stabilisation of the condition as a result of treatment with this drug; AND

The treatment must be the sole PBS-subsidised therapy for this condition; AND

Must be treated by a haematologist; or

Must be treated by a non-specialist medical physician who has consulted a haematologist on the patient's drug treatment details; AND

Patient must be at least 18 years of age.

The authority application must be made via the Online PBS Authorities System, or in writing via HPOS form upload or mail and must include:

(1) details of the proposed prescription(s); and

(2) a completed authority application form relevant to the indication and treatment phase (the latest version is located on the website specified in the Administrative Advice).

At the time of the authority application, details (result and date of result) of the following monitoring requirements must be provided on the first continuing authority application:

(i) Haemoglobin (g/L)

(ii) Platelets (x10⁹/L)

(iii) White Cell Count (x10⁹/L)

(iv) Reticulocytes (x10⁹/L)

(v) Neutrophils (x10⁹/L)

(vi) Granulocyte clone size (%)

(vii) Lactate Dehydrogenase (LDH)

(viii) the upper limit of normal (ULN) for LDH as quoted by the reporting laboratory

(ix) the LDH:ULN ratio (in figures, rounded to one decimal place)

Listing of Pharmaceutical Benefits (NHL) - Schedule 4 part 1

Paroxysmal nocturnal haemoglobinuria (PNH)

Transitioning from non-PBS to PBS-subsided treatment - Grandfather arrangements

Patient must have received non-PBS-subsidised treatment with this drug for this condition prior to 1 November 2025; AND

Patient must have a documented PNH granulocyte clone size equal to or greater than 10% within the 3 months prior to initiating non-PBS-subsidised treatment with this drug; AND

Patient must have experienced an inadequate response to a complement 5 (C5) inhibitor demonstrated by a haemoglobin level of less than 105 g/L prior to initiating non-PBS-subsidised treatment with this drug; or

Patient must be intolerant to C5 inhibitors as determined by the treating physician prior to initiating non-PBS-subsidised treatment with this drug; AND

Patient must have been receiving treatment with at least one C5 inhibitor for at least 3 months prior to initiating non-PBS-subsidised treatment with this drug unless intolerance of severity necessitating permanent treatment withdrawal had occurred; AND

The treatment must be the sole PBS-subsidised therapy for this condition; AND

Patient must have experienced clinical improvement as a result of treatment with this drug; or

Patient must have experienced a stabilisation of the condition as a result of treatment with this drug; AND

Must be treated by a haematologist; or

Must be treated by a non-specialist medical physician who has consulted a haematologist on the patient's drug treatment details; AND

Patient must be at least 18 years of age.

The authority application must be made via the Online PBS Authorities System, or in writing via HPOS form upload or mail and must include:

(1) details of the proposed prescription(s); and

(2) a completed authority application form relevant to the indication and treatment phase (the latest version is located on the website specified in the Administrative Advice).

At the time of the authority application, details (result and date of result) of the following monitoring requirements must be provided:

(i) Haemoglobin (g/L)

(ii) Platelets (x10⁹/L)

(iii) White Cell Count (x10⁹/L)

(iv) Reticulocytes (x10⁹/L)

(v) Neutrophils (x10⁹/L)

(vi) Granulocyte clone size (%)

(vii) Lactate Dehydrogenase (LDH)

(viii) the upper limit of normal (ULN) for LDH as quoted by the reporting laboratory

(ix) the LDH:ULN ratio (in figures, rounded to one decimal place)