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

Paroxysmal nocturnal haemoglobinuria (PNH)

First continuing treatment

Patient must have received PBS-subsidised treatment with this drug for this condition under 'Initial 1' (new untreated patient) or 'Initial 2' (switching from a Complement 5 (C5) inhibitor); 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, medical practitioners must request the appropriate number of vials for 4 weeks supply per dispensing as per the Product Information. A maximum of 5 repeats may be requested.

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)

Subsequent continuing treatment

Patient must have previously received PBS-subsidised treatment with this drug for this condition under the 'First continuing treatment', 'Return' criteria, or 'Grandfather' treatment restrictions; 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, medical practitioners must request the appropriate number of vials for 4 weeks supply per dispensing as per the Product Information. A maximum of 5 repeats may be requested.

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

Paroxysmal nocturnal haemoglobinuria (PNH)

Transitioning from non-PBS to PBS-subsidised treatment - Grandfather arrangement 1 (Complement 5 (C5) inhibitor inexperienced patients)

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

Patient must not have received any treatment for this condition prior to commencing non-PBS-subsidised treatment with this drug; AND

Patient must have had a diagnosis of PNH established by flow cytometry prior to commencing non-PBS-subsidised treatment with this drug; AND

Patient must have had PNH granulocyte clone size equal to or greater than 10% prior to commencing non-PBS-subsidised treatment with this drug; AND

Patient must have had a raised lactate dehydrogenase value at least 1.5 times the upper limit of normal prior to commencing non-PBS-subsidised treatment with this drug; AND

Patient must have experienced a thrombotic/embolic event which required anticoagulant therapy prior to commencing non-PBS-subsidised treatment with this drug; or

Patient must have been transfused with at least 4 units of red blood cells in the last 12 months prior to commencing non-PBS-subsidised treatment with this drug; or

Patient must have had chronic/recurrent anaemia, where causes other than haemolysis have been excluded, together with multiple haemoglobin measurements not exceeding 70 g/L in the absence of anaemia symptoms prior to commencing non-PBS-subsidised treatment with this drug; or

Patient must have had chronic/recurrent anaemia, where causes other than haemolysis have been excluded, together with multiple haemoglobin measurements not exceeding 100 g/L in addition to having anaemia symptoms prior to commencing non-PBS-subsidised treatment with this drug; or

Patient must have had debilitating shortness of breath/chest pain resulting in limitation of normal activity (New York Heart Association Class III) and/or established diagnosis of pulmonary arterial hypertension, where causes other than PNH have been excluded prior to commencing non-PBS-subsidised treatment with this drug; or

Patient must have had a history of renal insufficiency, demonstrated by an eGFR less than or equal to 60 mL/min/1.73m2, where causes other than PNH have been excluded prior to commencing non-PBS-subsidised treatment with this drug; or

Patient must have had recurrent episodes of severe pain requiring hospitalisation and/or narcotic analgesia, where causes other than PNH have been excluded prior to commencing non-PBS-subsidised 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)

At the time of the authority application, medical practitioners must request the appropriate number of vials for 4 weeks supply per dispensing as per the Product Information. A maximum of 5 repeats may be requested.

A patient may qualify for PBS-subsidised treatment under this restriction once only. For continuing PBS-subsidised treatment, a grandfathered patient must qualify under the Subsequent continuing treatment criteria.

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

Paroxysmal nocturnal haemoglobinuria (PNH)

Transitioning from non-PBS to PBS-subsidised treatment - Grandfather arrangement 2 (Complement 5 (C5) inhibitor experienced patients)

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

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

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

The treatment must have been in combination with one C5 inhibitor for a period of 4 weeks during initiation of therapy; 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)

At the time of the authority application, medical practitioners must request the appropriate number of vials for 4 weeks supply per dispensing as per the Product Information. A maximum of 5 repeats may be requested.

A patient may qualify for PBS-subsidised treatment under this restriction once only. For continuing PBS-subsidised treatment, a grandfathered patient must qualify under the Subsequent continuing treatment criteria.